All Episodes

#159: Emily Figueroa - Social Media Manager and Adult with SMA

#158: Alberto Lopez -A single father talks about raising his daughter with SMA

#157: Delphine Andrews - Life coach and disability advocate with SMA

#156: Maylan Chavez and Sory Rivera: Friends and podcast hosts with SMA

#155: Jasmine Jackson, digital creator and mother with SMA

#154: Coach Damon Vincent, adaptive fitness specialist

#153: Annie Heathcote, entrepreneur and speaker with SMA

#152: Kamil Goungor, Disability activist

#151: Anton Paras, marketing executive in digital health

#150: Jenna and Tanya Vega share their love story

#149: Jessica Keogh, Ed. D., Educator and Life Coach With SMA

#148: Ashley Fox and Madeline Engel discuss friendship and caregiving

#147: Brandi Lewis, rare disease advocate and speaker

#146: TJ Wall, graduate student with SMA

Carrie Manriquez, Mother of adult son with SMA

#144: Doug McCullough, Advocate and Author With SMA

#143: Cory Lee, Travel Blogger and Influencer With SMA

#142: Savannah and Will Huff, Interabled Couple

#141: Quinn Bucher, Theater Student With SMA

#140: Lucy Trevino, Academic and Advocate

#139: Dianna Warren, Ms. Wheelchair USA Ambassador

#138: Nolan and Blake Shofner, Brother Entrepreneurs

#137: Jared Wayland: Graphic Designer and Music Connoisseur

#136: Shaniqua Granby Discusses Identity and Community

#135: Ryan Kinnear, Fisherman and Hunter With SMA

#134: Dr. Edward Smith, Neurologist and SMA Specialist

#133: Rebecca Mulhall, Parent of Twins With SMA

#132: Collin Pollock, Business Owner With SMA

#131: Candis Welch, Disability Advocate and Ms. Wheelchair California

#130: Andrew Cherico, College Student With SMA

#129: Rylie Erbacher, Rare Artist With SMA

#128: Jenna Coburn, Social Media Manager, LGBTQIA+ and Disability Advocate

#127: Kevan Chandler, author, traveler, and nonprofit founder

#126: Janelle Fiesta and Antoine Vuong, interabled couple

#125: Brooklyn Nichols, child author with SMA, and her mother, Keanna

#124: A conversation with an interabled couple, Steve and Brittany Bingman

#123: A Conversation With LaMondre Pough and Gabrielle Runyon, Two Black Disability Advocates

#122: A Conversation With Rare Disease Advocates

SMA PODCAST 121

#120: Dom Evans, Activist and Filmmaker

#119: Chaz Hayden Discusses His Debut Novel

#118: Aging and SMA

#117: Delphine Andrews and Kevin Davis, Interabled Couple

SMA PODCAST 116

#115: Jose Flores, Author and Motivational Speaker

#114: Daniele Johnson, SMA Mom and Advocate

#113: Dustin Swafford, Power Soccer Coach and Player

#112: Lexi Villa, Social Media Influencer and Disability Advocate

#111: Steven Verdile, Graphic Designer and Disabled Creative

#110: Author Ben Mattlin Discusses Disability Pride Month

#109: 2022 Cure SMA Conference Recap

#108: A Mother Discusses the Approval of Evrysdi for Infants With SMA

#107: A Conversation About Mental Health With LGBTQ+ and Disability Advocates

#106: A Conversation About Women’s Mental Health With Rare Disease and Disability Advocates

#105: A Conversation About Men’s Mental Health With Rare Disease and Disability Advocates

#104: Interview With Ben Lou, Mathematics Student With SMA

#103: Discussion on Clinical Trials and Advocacy

#102: Interview with Amber-Joi Watkins, SMA Mom and Advocate

Using Ultrasound May Help in SBMA Diagnosis & Dealing With Hospital Stays

#101: Interview with John Milligan, Asuragen Scientist & SMA Testing Expert

SMA Screening Now Available to 87% of Newborns in US & Olympics Lacked Disability Representation

Evrysdi for Infants Under 2 Months Old Given FDA Priority Review & Adapting to a New Wheelchair

#100: Interview with Gabrielle Runyon, College Student with SMA and Disability Advocate

SMA Linked to “Significant” Economic Burden

Therapy Effectively Treats SMA in Mice With Evident Symptoms & Tackling New Challenges With SMA

#99: SMA and Motherhood

Spinraza Plus Zolgensma Offers Little Extra Benefit in Type 1, Study Suggests

Spine Surgery Effective in SMA Type 1 Children & Mentally Preparing For Surgery

Wheelchair Hockey Linked to Physical, Psychological Gains & Preserving Privacy When Living with SMA

Scholar Rock Shares Design of Phase 3 Trial Testing of Apitegromab & Staying Organized With SMA

#98: How to Manage Caregivers, a Chat with SMA Contributors DeAnn Runge and Michael Morale

Spinraza May Restore Development of Motor Neurons & Why Finding Reliable SMA Carers Is Not Easy

Son With SMA Inspires Character on ‘Ordinary Joe’ TV Show & How SMA Can Shape Creativity

Evrysdi May Be Available in Early 2022 to Eligible UK Patients &Modifying a Wheelchair for SMA Needs

Spinraza Improves Hand Dexterity in SMA Type 2 Children & Achieving Ambitions with SMA

#97: Interview with Ali Ramos, a Social Worker and Disability and LGBTQ+ Activist.

Spinraza Delays Do Not Directly Affect Children in Italy & Making Goals with SMA

Saliva Samples May Help Diagnose SMA & Moving Into a New Home with SMA

Poor Spinraza Adherence Leads to More Illness, Healthcare Costs & December Can Be Bittersweet

Treatment Delays May Be Less Important Than Family Support & Cherishing Togetherness During Holidays

Phase 3 Trial of Apitegromab on Track for This Year & Managing Technological Issues With SMA

Neurofilaments and Nerve-muscle Test Show SMA Severity & SMA Adults Struggle to Access Evrysdi

#96: Chat with SMA Columnist Halsey Blocher and her Mother & Caregiver, Heather Dye

Novartis Applauds Move to Reimburse Families for Zolgensma & Making Connections with SMA

Acute Liver Failure Warning Added to Zolgensma Label & Time Management and SMA

Oral SMA Therapy Is Ideal and Aids Motor Skills and Breathing & Why No Changes in SMA is Good

TXA Reduces Blood Loss During Scoliosis Surgery & Taking A Break For Emotional Wellness

#95: Interview with the SMA Creatives Behind "Spaces" Music Video

Weak Trunk Muscles And Spine Problems Need Early Attention in SMA & The Human Side of the Internet

Clinical Trial to Test Antibody Use With Evrysdi & Thanksgiving Challenges

Organization Aims to Make Planes Wheelchair-Accessible & Dealing with Accessibility Issues at Home

New Spinraza Delivery Technique for Patients & How To Refer to Someone With a Disability

#94: Interview With Pamela K. Muhammad, Author and Entrepreneur With SMA

Anxiety and Depression Common Among School-Age SMA Patients in China & Facing SMA Challenges

Belgian Newborn Screening Pilot for SMA Becomes Official & Getting Through Physical Therapy with SMA

SMA Doesn't Stop Teen From Pursuing Academic Dreams & How To Make A More Accessible World

Cure SMA Advocates for Better Wheelchair Storage for Air Travel & SMA Issues Can Be Overwhelming

#93: Interview With Garrett Lerner, Co-creator and Executive Producer of NBC Series “Ordinary Joe”

Canadian Agency Favors Zolgensma Only for Babies Up to 6 Months Old & Sometimes SMA Is Just Too Much

Phase 3 Apitegromab Trial Planned for Non-ambulatory Types 2 And 3 & Latest Topics on Our Forums

Neurofilament Blood Levels Likely to Predict Treatment Response in SMA

Quality of Life Survey Aims to Help Inform SMA Community Needs & Situations That Could’ve Gone Wrong

#92: Discussing Disability and Employment

Pre-symptomatic Infants Retain Swallowing Ability in Evrysdi Trial

Spinraza RESPOND Trial Enrolling Children Not Helped by Zolgensma

Low Bone Density Puts SMA Children in China at Risk for Fractures & DeAnn Runge's Favorite Hobbies

Certain Abilities Decline in SMA Children Unable to Walk & Gaming to Find Accessibility

#91: Blake Watson, Web Designer and Developer

Trial of Spinraza at High Dose for Patients Who Have Used Evrysdi & Dealing With Your Period

Blood NfL Levels May Mark SMA Severity, Therapy Efficacy in Very Young

Variant in Androgen Receptor Might Be Useful in Treating SBMA

Cure SMA And Cytokinetics Renew Joint Efforts to Raise Funds and Awareness

#90: Interview With Judy Heumann, Disability Rights Activist

SMA Caused by Mutation in ASAH1 Gene Reported in Romania

2 SMN2-targeting Therapies Work Better Than 1 in Mouse Model

Indigenous Groups in Canada May Have Highest Rate of SBMA in World

Mothers Want to Improve Children’s Quality of Life & Making Friends as an Adult With SMA

#89: Interview with Carole St-Laurent, A Children’s Author With SMA

$1M Raised for ‘SpawnTogether,’ Disabled Gamers Project & DeAnn's Jaco Robotic Arm

Some SMA Patients Unable to Walk Unassisted May Have Trouble Chewing

Behind-the-Scenes of #31DaysofSMA

SMA UK Network Helps Patients Navigate Adulthood

Most Parents Surveyed in Japan Support SMA Newborn Screening

SMA Type 1 Affects Sensory Nerves as Children Age, Small Study Shows

‘Best Possible Outcome’ for SMA? Newborn Screening, Then Zolgensma

Zolgensma Helps Mobility of SMA Children in Qatari Real-world Study

FDA Lifts Hold on Clinical Trials of Intrathecal OAV-101 & Self-Acceptance and Rejecting Ableism

#88: SMA Awareness Month & 31 Days of SMA 2021

Zolgensma-Evrysdi Combo Likely Beneficial for SMA Type 1

Inviting People to Sit at the Table of My Disabled Life & 31 Days of SMA Thus Far

Muscular Dystrophy Canada Funds Projects on Newborn SMA Screening

Spinraza Improves Lung Function in SMA Type 2 Child, Report Says

Why My Journey With Disability Pride Isn’t Linear & 31 Days of SMA Initiative

Novartis Stopping Work on Branaplam as Oral SMA Therapy & 31 Days of SMA

#87: Interviews with Allie Williams, Vocal Coach and Disability Advocate

Early Work Supports Apitegromab’s Safety as SMA Muscle Therapy & Jaco Robotic Arm Approval

SadBaby Crowdfunding Aims to Help SMA Families & Accessibility, Disability Pride and Success

Partnership Aims to Lower Out-of-Pocket Costs for Rare Disease Meds

‘Wearable Cyborg’ Helps Improve Walking Ability And Muscle Strength

VRK1 Mutations Were Found in Two Adult-Onset SMA Hispanics & the SSI Restoration Act of 2021

COVID-19 Infection Turns Severe in SMA Type 1 Child & Treating Your Body Right

#86: Price Wooldridge, The Voice Behind the SMA Flash Briefings

Sma Treatment May Be More Effective by Altering an Underlying Molecule & Having a Service Dog

Spinraza Improves Motor Function in Children And Adults With SMA Type 3 Children

Stem Cell Therapy May Benefit Some Type 1 Infants & Disability Pride Month

People in UK Urged to Sign Petition Adding SMA to Newborn Screening

US Pediatricians Often Overlook Steps in Diagnosing SMA, Surveys Show

Texas Adds SMA to Its Newborn Screening Program

#85: Interview With Mikey and Noelle Hazel, Siblings with SMA

Japan Approves Evrysdi as First At-home, Oral SMA Treatment

Major Developmental Milestones Met in Infants, Zolgensma Data Show

Newborn Screening in Massachusetts Seen as Highly Accurate And Effective

Spinraza Leads to Better Swallowing, Farther Walking & Weekly Wins: Sharing Wins in Our Forums

Apitegromab Safely Increases Motor Abilities in SMA Types 2, 3

Evrysdi Linked to Improved Motor Function in 2 Trials & Finding Community Support

SMA Added to North Carolina’s Newborn Screening Program

NICE Favors Zolgensma Being Added to England’s Public Health Program

Navigating the Past Year Without Caregivers Has Been Challenging

#84: Scholar Rock Updates and Community Highlights

NICE Initially Against Adding Evrysdi to UK Public Health System

SMA and Sibling Relationships: A Brother’s Perspective & Sticking With Evrysdi

Asuragen’s Lab Test Can Speed Up Diagnosis of SMA Carriers and Patients

Stress-induced Protein May Serve as New SMA Biomarker in Infants

Apitegromab, Muscle-directed Therapy for SMA, Put on FDA Fast Track

SMN2 Copy Number and Rare Variant Influence SMA Severity

#83: Interview with Christine Getman and Scottie Foertmeyer, Couple and Magic Wheelchair Executives

SMA May Carry Higher Risk of Fluid Buildup in The Brain & Reflections on Memorial Day

Apitegromab Safely Counters a Muscle Growth Suppressor in Patients With SMA

Cure SMA Offers US Patients Free Tools to Boost Their Independence & Managing Fatigue With SMA

Remembering to Prioritize Mental Health & Self-Esteem, Post Vaccination Plans and More

Neurofilaments May Not Be Biomarker for Older Patients & The New CDC Mask Recommendations

Warning of TMA, Rare Blood Disorder, Added to Zolgensma’s Safety Label & Friendships and SMA

#82: Interview with Hawken Miller, Journalist with Muscular Dystrophy

Sex And The Number of Copies of The SMN 2 Gene Can Influence Age of Onset for SMA Type 3 Patients

UK NICE Expands Access to Spinraza for SMA Type 3 & The Disappointment of a Jaco Robotic Arm Denial

German Study Urges Newborn SMA Screening, Citing Better Outcomes & Managing Chaos

Grant Awarded to Find Affordable Treatments for Children & SMA Would You Rather

Novartis Poised for Phase 3b SMART Study of Zolgensma & SMA Adventures

Danish Family Races to Raise $2.4M for Daughter’s Zolgensma Therapy & Staying True To Yourself

#81: Interview With Chaz Hayden - Writer and YouTuber with SMA

#AANAM – Zolgensma May Lead to Faster, Greater Gains Than Spinraza

Neurofilaments Before Spinraza May Predict Motor Improvements & Reviewing The Sound of Metal

Health Canada Approves Evrysdi for At-home Treatment

Immune Response to Zolgensma Can Be Common in Older SMA Children

Prenatal Enrollment Yields Faster Results from Newborn Screening

Apitegromab Improving or Stabilizing Motor Function in Children And Young Adults With Small

#80: Updates on Apitegromab and Discussing SMA Columns

European Alliance Calls for Newborn Screening & Allergy Season, Sleep Solutions and SMA Treatments

Architecture Of The SMN Complex Is Altered In People With SMA & Comparing Wheel Drive Wheelchairs

Spinal Fluid Changes With Spinraza’s Use Mild, Possibly Due to Lumbar Puncture

Evrysdi Approved in Europe as First Oral, At-home Treatment & The Evrysdi Chronicles

Benefits of SMA Newborn Screening Outweigh Disadvantages In New Survey & Everyday Struggles With SMA

#79: Interview With Ainaa Farhanah, a Graphic Designer Living with SMA

Scholar Rock Wins US Patent for Apitegromab & Receiving The COVID-19 Vaccine And Getting Outside

Zolgensma Helped Pre-symptomatic Babies Achieve Age-Appropriate Motor Milestones

Zolgensma Continues to Prevent Motor Function Decline for 5 Years

Evrysdi Leads to Longer-term Benefits for SMA Types 2, 3, Data Show

Zolgenzma Found To Be Safe And Effective in SMA Toddlers & The Importance of Schedules And Routines

Evrysdi Safe in SMA Patients Previously Given Other Therapies & The Evolution of Technology

SMA Foundation and PTC Joint Efforts to Fund Regenerative Medicine Research

Reldesemtiv Aids Motor and Respiratory Strength in Types 2 and 3, Early Trial Finds

Zolgensma Available Soon to Eligible SMA Type 1 Patients in England, Scotland

Muscles Controlling Eye Movements Not Affected by SMA & Never Give Up Searching for Your Advocates

78: Discussing Switching Treatments, News Stories, and Recent Columns

The Challenges Of Life as a Teen And Young Adult With SMA & Being Denied Access to SMA Treatment

CHMP Favors EU Approval of Evrysdi as 1st Oral, At-home SMA Treatment & Discussing GI Issues

Evrysdi Seen to Improve Survival, Motor Development of Infants with SMA Type 1

Mother Raises 4 Adopted Girls From China With SMA, Chronic Illnesses

Brain Involvement in SMA Type 1 Still Poorly Understood & Struggling to Personalize New Wheelchair

Study Finds Varying Perspectives From Parents on Spinraza Therapy for Children

#77: Interview With Jeremy Camp, SMA Parent and Advocate

New SMN-boosting Molecule Shows Promise as Add-on Therapy & Rare Disease Day And Forums Topics

Rare Case of SMA Linked With Myoclonic Epilepsy Detailed in Report

Assistive Devices Should Be Standard in Managing SMA Type 1 & I’m Grateful for My Friends

Quantitative MRI Is Sensitive Measure of Muscle Decline

Spinraza Improves Motor Function in Case of Teenager with Late-onset SMA

A ‘Miracle’: Spinraza Gives Boy a Chance to Grow Up & Life Is an Adventure, So Let’s Be Pioneers

#76: Discussing Treatment Updates, Vaccine Access, and Recent Columns

EMBRACE Trial Supports Spinraza’s Benefits in Broad Range of SMA Patients

NORD’s 6th ‘State Report Card’ Notes Progress, Raises Concerns

Rare Disease Groups, Pharmas Join SMA Europe Push for Newborn Screening

Motor Function, Breathing Important in Choosing Treatment

More Severe COVID-19 Symptoms Seen in Children with SMA Type 1

Better Ways of Capturing Progression in Types 2 and 3 Identified in Study

#75: Alvaro Cheherlian, Founder and CEO of Wrekt Svpply

Cure SMA Booklet Outlines Possible Risks, Benefits of Combining Treatments

TOPAZ Trial Results on Muscle Therapy Likely by June, Scholar Rock Says

Resolutions, Mindsets and Emotions & Transitioning SMA Patients From Older To Newer Wheelchairs

Online for 2021, Team Cure SMA Race Series Planning Variety of Events

Zolgensma Approved to Treat Young SMA Patients in Canada

Keeping Your Child with SMA in School During the Cold and Flu Season & Soaring With Hope

#74: Interview with Katie Napiwocki and Andy Rusch, Interabled Couple

New Cure SMA Webinar Series to Focus on Health, Wellness & Let's Talk About Sex and SMA

Higher-dose Spinraza Trial Now Enrolling Part B After No Safety Issues Found

This Year, I Want to Be Brave & The Challenges That Come With Pursuing Independence

Informal SMA Caregivers Surveyed in Europe Report High Daily Burden & Relevant Topics On The Forums

Zolgensma Linked in 3 Cases to Serious But Treatable Blood Disorder

Zolgensma Approved to Treat Young SMA Patients in Canada & Kidney Stone And a Trip To The ER

#73: Interview With Tyler Dykema, Artist and Musician with SMA

Paramedian Injection Approach Found to Ease Spinraza Administration & Difficult On Getting Evrysdi

Ella Has Fun While Sheltering at Home & Stepping Into a New Me in the Year Ahead

Being An Uncle With SMA and How This Role Can Change One's Life

Smart Tech Is Integral to My Quality of Life & 2020 Highlights

Rising From the Ashes and Flying With Broken Wings & Finding My Voice In the Disability Community

Financial Burden of SMA Much Higher Than Other Conditions & The Mental Benefits of Physical Therapy

Measuring Neurological Impact of Polio Proves Useful in SMA, Study Reports

Genetic Analysis of Families Finds Novel Mutations in SMA & The Lack Of Privacy When Living With SMA

Zolgensma-associated Liver Abnormalities Common but Manageable & I’m Disappointed, and That’s OK

#72: Evrysdi, Physical and Mental Health, and Highlights of 2020

Neurological Alliance Report Calls for Better Treatment, Care for UK Patients

Spinraza Linked to Temporary Abnormalities in Immune Cells in 2 SMA Infants

Extensive Trial Monitoring Confirms Evrysdi Does Not Damage Vision & The Series "Boy Meets World"

Olesoxime Failed to Benefit SMA Type 2 and 3 Patients, Final Trial Data Show

Gene Therapy Given Directly to Spinal Canal Might Be Safer With ‘Silencing’ Step

Exploring The Intersection of Identity and SMA

#71: Interview with Kristen Resendez, SMA Parent and Advocate

Spinraza Shown to Preserve Respiratory Muscle Strength in SMA Type 2 Patients

Lung Health Declines in Step With Motor Loss in Types 2 and 3, Study Finds

Reflecting On The Insanity of 2020 and The People Who Got Us Through This Year

Spinraza’s Benefits Mild, Transient in Infant With Severe Type 0 SMA

Molecule Similar to Evrysdi But Possibly Safe at Higher Doses Identified

How An Urology Appointment Turned Into A Fiasco & Typing Strategies, Vitamin D and Friendsgiving

#70 - Discussing the JACO Robotic Arm with Ron Borgschulte

Muscle MRI Captures Spinraza’s Effects on Tissue Fibers in Type 3 Brothers

NICE Reviews UK Spinraza Reimbursement for SMA Type 3 Patients Unable to Walk

The Importance Of Balance While Maneuvering On Uneven Surfaces For People With SMA

Muscle-directed Therapy SRK-015 Improves Motor Function in SMA Types 2 and 3 & Hibernation in Winter

Evrysdi Approved in Brazil for Spinal Muscular Atrophy (SMA)

Spending Time In & Out Of You Chair So You Give Your Body The Balance It Requires & Managing Stress

#69 - Interview With Lamondre Pough

PAN Foundation Offers Financial Help for SMA Treatment & Routine Changes As Winter Sets In

Adults With SMA Who Sought Specialty Care After Spinraza Approval May Have More Severe Disease

Embracing The Imaginative Spirit of Halloween & Cold Feet, JACO Robotic Arm and Caregivers

‘Think 3 at 3 Months’ Drive Seeks to Raise Awareness of Infant Movement Milestones & PCA Services

SMA Type 3 Adults May Have Issues Regulating Component of Cell’s ‘Skeleton,’ Study Suggests

How Memories Of a Dying Shopping Mall Made Kevin Schaefer About Adapting to Change

# 68 - October Roundtable Discussion

4-aminopyridine Fails to Improve Muscle Function in SMA Type 3 Patients & New Service Dog

SMA Type 1 Infants Treated With Zolgensma Achieving Milestones, Data Show

Spontaneous Trips, Reading, Socially Distant Celebrations, Delayed Deliveries & More

Infants Receiving Evrysdi Continue to Improve and Achieve Motor Milestones

Novartis to Open, at FDA Request, New Trial of Zolgensma for Older Patients

How SMA Affects Your Ability to Draw, Treasuring Little Things & Setting Boundaries With Caregivers

#67 - Interview With Maylan Chavez

Spinraza Leads To Motor Gains Over Time in SMA Type 3 Adults

GridPad Trilogy Communication Device Aids People With Speech And Motor Disorders Who Have SMA

Kevin Schaefer Talks About How The Past Few Months Have Been And How He’s Adapted to a New Normal

Electric Bike Technologies Donates Liberty Trikes To Aid The Mobility Of Children With SMA

AveXis Now Known as Novartis Gene Therapies, Focus of Continuing Work

Eye Doctor Appointment Adventures, How Everything Takes Extra Effort In The World Of SMA & More

NICE Widens Its Zolgensma Appraisal Due to European Marketing Authorization

Activities Underway for Newborn Screening Awareness Month

Experiences With a BiPAP Machine And The Pros And Cons of Wearing One & Wardrobe Challenges

#66: Talking About Evrysdi, SRK-015, and Recent Columns

Nerve-Muscle Molecule May Be an SBMA Therapeutic Target & Getting a New Wheelchair and a Service Dog

Ohio Study Finds Zolgensma Safe and Effective, Particularly in Younger Infants

Comparing The Fictional Superhero Team The X-Men To The SMA Community & Getting Back To School

FDA Grants Rare Pediatric Disease Designation to SRK-015 for SMA & Going to The Bathroom With SMA

SMA Registries Offer “Real-World Experience” That Makes The Best Treatment Possible

A Digestive Dilemma & Some Of The Final Stories Featured in The 31 Days of SMA Campaign

#65 - Interview With Dwight Reed

Muscle and Combo Therapies Likely Next Focus for SMA & DeAnn Runge's 3 Year Journey With Spinraza

SMA Debate: Might Systemic Treatment Be Best?

31 Days of SMA Story: Resilience and SMA

Roundtable Podcast Discussion on Technology

61 Year Old With SMA Type 3 Says With Evrysdi “Every Day is Good”

Evrysdi and Spinraza Target The SMN2 Gene, But in Different Ways

The Joy of Always Learning, Evrysdi (risdiplam) & Two Different Perspectives on Education

The Expertise in RNA Biology at The Core of PTC’s Formative Work with Evrysdi

Evrysdi Has Parents “Totally Optimistic” For The Future of Their Boy With SMA Type 2

Frequently Asked Questions About Evrysdi (Risdiplam) & Great Stories From 31 Days of SMA

FDA Approves Risdiplam, Now Known as Evrysdi, The First Oral Treatment For All SMA Types

Plans Advance for Biomarkers Panel to Assess Drug-induced Skeletal Muscle Injury

#63 - Interview With Hugo Trevino

The First Few Days of The 31 Days of SMA Initiative & A Story On Dating and Disability

Greatest Loss of Motor Skills at Ages 5 to 13 In SMA Type 2 Patients & Wheelchair Evaluation

Biogen is Planning to Launch a First Trial, Testing Spinraza in Children Previously Given Zolgensma

Thinking Ahead, Disability vs. Disease & Diverse Reading Selections

Iowa Adds SMA as a Pilot Program For Newborn Screening & DeAnn Runge Got a New Kitten

Spinraza Eases Fatigue In Adults With SMA But That Benefit Wanes

Socially Distanced Visits & 31 Days of SMA

#62 - Interview With Shawn Stewart

SMN Protein Levels in Blood May Mark SMA Severity & Disability Pride Month

How New Glia Cell Markers May Provide Insight Into Neuromuscular Diseases, Including SMA and ALS

Embracing Disability in Pride Month, Tips to Beat The Summer Heat & Travel and Equipment Rental

The Significant Limits to Treatment Seen For a Spinal Muscular Atrophy, Type 0, Baby

Disparities Found in Parents And Children’s Perceptions of Spinal Muscular Atrophy

How Having SMA Has Led To Many Positive Interactions With Strangers, Returning to School & Playlists

#61 - Recapping the 2020 Cure SMA Virtual Conference

Motor Function Improvement After One Year of Spinraza in Children With SMA Types 1-2

Systemic Treatment May Be More Effective For Spinal Muscular Atrophy

Switching Treatments, Summer Reading & Dealing With Health Issues On Top Of SMA

Risdiplam Continues To Show Promise For Treating SMA & Pulling Off a Surprise

31 Days of SMA & How 96% of Children Given Spinraza As Newborns Are Able To Walk According To Trial

SMA My Way: A New Platform and Patient-Focused Community & What to Binge-Watch on Netflix

Interviews Probe How Unaffected Siblings Learn Genetic Implications of SMA

Too Little is Known About The Care Needs of Adults With SMA

The First Virtual Cure SMA Conference, Creating a Sanctuary, Scoliosis & Epic Wheelchair Stories

#60 - Interview With George Corbin

How Heart Problems in SMA May Be Tied To Calcium Dysregulation

SHINE Study Data Finds Spinraza Shows Sustained Efficacy Over Years of Use

Cure SMA Conference Going Virtual in 2020 & Tips On Purchasing A New Electric Wheelchair

Feeding Issues Are Still Common Among SMA Type 1 Infants, Despite New Therapy Options

Real-World Study Shows That Spinraza Meets Most Therapeutic Expectations of Adult SMA Patients

#58 - A Conversation with Brianna Albers

Tips For Staying Active and Mentally Engaged This Summer & How The JACO Robotic Arm Opens Doors

Study Finds Respiratory Weakness in SMA is Most Pronounced in Childhood & Treatment Options for SMA

SMA Treatment and Screening Programs “Eessential” in The Covid-19 Pandemic

Risdiplam, Switching Treatments & Difficult Breathing While Wearing Masks

Study Addresses Dilemmas Regarding Newborn Screening and SMA Treatment

How a Family and Doctor “Partnership” That Led to a Baby’s Spinraza Treatment

Fatigue Experienced Prior Spinraza Injection & Cure SMA COVID-19 Care Package

#58 - How COVID-19 Affects Spinraza and Other Medical Appointments

How Lung Ultrasound is a Reliable Option to Chest X-rays for Monitoring Children

Study Suggests The Interplay of Motor Neurons and Glial Cells is At The Root of SMA

Differences Between Risdiplam and SRK-015, Dealing With Anxiety & Taking Things For Granted

How Risdiplam Defies SMA “Natural History” in Type 1 Infants, According to FIREFISH Study Data

How Growth-Friendly Spinal Implants May Help SMA Children Before Fusion Surgery

How SMA-Related Humor Presents Itself in Unexpected Moments, Fundraising Options & Online Therapy

#57 - Interview with Heather Kerstetter

How Taking Part in Adapted Sports Improves Mental Health in Patients With SMA

Discussing The Lack of Muscle Cell Signaling Which is Seen to Kill Motor Neurons in SMA-LED2

Telemedicine, Therapy Platforms, Alternative Exercise Routines & Hospital Preparedness Folders

Discussing Motor Gains and Safety with Zolgensma’s IT Use in a STRONG Trial

First Patient Treated in The Phase 2/3 Trial, Assessing Higher Doses of Spinraza

Playing Phone Games and Nintendo Switch & Home Exercise Routines and Robotic Assistance Devices

#56 - SMA Treatments Overview and “Crip Camp” Discussion

How Adult Patients with SMA Benefit From Spinraza Treatments

Ideas for Staying Fit and Healthy During Isolation & Approval of Zolgensma for Treating SMA in Japan

Accessible Toys, Having Fun With Subscription Boxes & Kevin Schaefer's Obsession With Action Figures

Odd Jobs, Spring Cleaning & French Study Finds Improvements in Palliative Care for SMA Type 1 Babies

FDA Shifts Risdiplam Decision to August to Include Data on Older Patients

Managing Spinraza Treatments, Caregivers, Outside Deliveries and More During COVID-19

#55 - Interview With Victor Guerra

How Scoliosis Surgery in SMA Children is Linked to Permanent Motor Skill Loss

Discussing Markers of Inflammation Essential for AAV Gene Therapy Use & Thoughts About “Crip Camp"

The SMA News Today Forums: a Place to Share Ideas That Will Brighten Spirits During Difficult Times

Global Rare Disease Group’s Goal to Have 1,000 New Therapies by 2027 & Benefits of a Service Dog

Book, Movie and Spotify Recommendations & Study Recommends a Lower Radiation Dose for Spinraza Scans

Alternative Solutions to Physical Therapy During the COVID-19 Pandemic

New Test Screens 420 Genes for Markers of SMA & A Message to Friends, Family About COVID-19

Tips For Self-Isolation During the Coronavirus & SYT13 Gene Can Prolong Life in Mice With SMA

Coronavirus (COVID-19): Precautions, Protecting Yourself and Staying Productive During Lockdown

#54 - Roundtable About Corona Virus

Risdiplam Under FDA Review & How the Lack of SMA Protein May Also Directly Contribute to SMA

Importance of Physical Therapy & Institute for Gene Therapies to Modernize Reimbursement Framework

COVID19, Spinraza, Bloating, SPC’s, Being Spontaneous & Transferring Out Of Wheelchair To Take a Nap

Being Spontaneous With SMA & How Body Composition May be a Biomarker of Motor Function in SMA

Maintaining Long-Distance Friendships & MDA Executive’s Discussion of Gene Therapies

Celebrating Rare Disease Day 2020 with #WhatMakesMeRareSMA & How Music Plays a Big Part in Life

#53 - Interview with Alyssa Silva

Public Interactions with People With Disabilities & Muscular Dystrophy Association Helping Others

Risdiplam Success in Treating SMA Type 1 Babies in FIREFISH Study & Kevin's Spinraza Injection

Talking About Rare Disease Day & Accessible Parking, Sleepovers with SMA and Product Reviews

The Discovery of a Potential New SMA Genetic Modifier & Having a Great Deal of Strength

How a CSF Protein Profile May Help Predict Spinraza Responses in Late-Onset SMA Patients

Kevin Schaefer Reads His Week’s Motivational Post & Katie Napiwocki's Comeback as a Columnist

52 - Luisa Palazola and Rare Disease Day 2020

How Magnetic Rods May Help Scoliosis in Children with SMA & Living With Caution, Not Fear

Early Access Program From Roche for SMA Therapy Risdiplam in Europe

Accessible Travel, Becoming Friends With Your Caregivers and The Importance of Exercise With SMA

New Treatment Algorithm For SMA Infants Via Vital Newborn Screening & Going To The Bathroom

ZPR1 Protein May Be New PotentialTherapeutic Target For SMA & NBC's Pilot Featuring Kid With SMA

Embracing The Power of Your Voice & Struggling As a Family

#51: Interview with Ryan Manriquez and Nora Zade

100 Free Zolgensma Treatments Worldwide in 2020 & Unplugging the iPad, Waste of Time

Adults With SMA Tend To Report Low Prevalence of Non-Motor Symptoms & Reading From the SMA Forums

Managing Stomach Issues As SMA Patient & Great Conversation in the SMA News Today Forums

Regular CSF Exams Are Urged Following Spinraza Treatments & Smart Home Devices

HHS Secretary's Efforts To Cure Rare Diseases & Taking Advantage of New Opportunities

Being Rare and Resilient & No Topic Is Off Limits on The SMA News Today's Forums

#50 - Roundtable Discussion - January 2020

Spinraza Stabilizes or Improves Motor Function in Older SMA Patients & The Reality of Aging

Multidisciplinary Approach Improves Spinraza Dosing Process in SMA Patients & Parenting and SMA

Importance of Media Representation for People With Disabilities & Conversations in the SMA Forums

Targeted Genetic Screening Helps Diagnosing Patients With SMA-Like Symptoms & Pranking With SMA

Early Trial Data Shows SRK-015 Increases Myostatin Growth Factor Levels in SMA Patients

Volunteering As A Reward & Humorous Interactions With People

#49 - Interview With Ashley Fox

Asuragen States New Screening Kit Analyzes SMN1 and SMN2 Genes in the Lab & Being Grateful

Risdiplam Improves Motor Function for SMA types 2 and 3 Patients & New Year's Resolutions

AveXis Unaware of the Cause of Inflammation that Led to the STRONG Trial Hold & 2019 Highlights

Kevin's JACO robotic arm & Pondering Possibilities of the Future

#48: Roundtable Discussion December 2019

FDA Grants Priority Review For Risdiplam & Going To Concerts

BillionToOne's Prenatal Blood Test Now Available in 4 Countries in Europe & Being Proactive With SMA

Movies That Teach You How To Live With SMA & Forums Recap

Exercise Helps Increasing Functional SMN Protein & Being Sick During Holidays

MRI Fiber Tracking May Be Potential SMA Biomarker for Response to Therapy & Thoughs On Risdiplam

Dealing With Assumptions About Physical Capabilities

#10yearchallenge & Risdiplam Getting Fast Tracked For Approval

#47 - Interview With James Ian

MRI Scans of Facial Nerves Can Help Diagnose SMA & Pets Teach Responsibility

New Potential SMA Therapeutic Target Found In Muscle Cells & Expressing Gratitude

How Rare Disease Family Goes Beyond the SMA Community & Personal Stories About SMA

Study Suggests SMA Patients May Benefit From A Low-Fat Diet & Dealing With Disappointment

SMN Protein Study Emphasises Importance of Early SMA Treatment In Newborn Screening

The Importance of Rejuvenation for People With SMA & The SMA Forums Highlights

#46 Roundtable Discussion - Discussion for November 2019

Study Suggests Spinraza Motor And Lung Functions Is SMA Type 3 Adults & Multitude Of Caregivers

Zolgensma Produces Promising Results In Young Children With Type II SMA & Life Progression

Having a High Quality of Life With SMA & Five Servings of Strength by Michael Casten

Scholar Rock’s Early Data Supports the Development of SRK-015 For SMA Types 2 and 3

Risdiplam Continues to Show Survival and Motor Function Improvements

SMA News And Perspectives 11 - 06 - 19

#45 - Interview With Jim and Becky Berry

#45 - Interview With Jim And Becky Berry

FDA Placed A Partial Hold On STRONG Trial Of AVXS-101 & Binge Watch TV Series

SMA STAT Genetic Test Is Enabling Earlier Diagnosis & Employment and SMA

Embracing The Imaginative Spirit of Halloween & SMA News Today Forums

Skye, The First Baby Treated With Zolgensma, Is Getting Stronger & Embracing Halloween

Oklahoma Ranks The Lowest On Programs That Are Key To Rare Diseases & Recent Columns

SMA News And Perspectives 10 - 23 - 19

Rare Disease Groups Seeking Public Support To Renew Newborn Screening Act & Winter Hibernation

Baby Girl's Family Brought From The UK to Ohio For Zolgensma Treatment & Maid of Honor

Overview Of The SMA Forums And Recent Discussions & Coming-Of-Age Stories And Living With SMA

#44 - Roundtable Discussion - Discussion for October 2019

Teen With Epilepsy Described As Rare Case of SMA & Alternative Options For Seating

Additional Space For Zolgensma Production With AveXis Catalent Partnership & Trending Topics

Problems With Metabolising Fat Molecules Are Worrisome In SMA Children & Being Inspirational

Risdiplam Is Raising SMN Levels In Older Patients In Durable Ways & Dealing With Disappointment

Adding Reldesemtiv to Spinraza Improved Muscle Function In An SMA Mouse Model & Finding Humor

Prenatal Blood Test For SMA Is Now Available & "Rolling Through The Seasons"

#43 - Interview With Jeff Olander

Macedonian Gaucher Activist For Rare Disease Patients & Getting Ready For The Cold

European Spinraza Label Updated to Reflect Long-Term Benefits & Trending Topics

High Chance Of Scoliosis Surgery in Types 1C and 2 & Superheroes With Disabilities

Spinraza Gives Babies With Type 1 SMA Hope For a Healthy Life in North Macedonia

Phase 2 Trial Shows Presymptomatic Spinraza Treatment to SMA Children Reaching Milestones

The World’s First Alport Stamp is a Macedonian Mom’s Latest Win for Rare Disease Patients

#42 - Interview With Kyle Harris

RNA Therapy Delivered by Viral Vector May Hold Potential to Treat SMA

Why Screening Tests For Zolgernsma’s Use Are Necessary & Motivational Post

SMA Candidate SRK-015 Shows Promise in Healthy Volunteers & More Independence

Study Says Children With SMA Are at Risk of Weak Bones And Fractures & More Independence

How AveXis’ OneGene Program Could Help Families Wanting Zolgensma & Trending Topics

Future Work Into Zolgensma, Gene Therapy And Other SMA Treatments & Physical Books

#41 - Roundtable Discussion - Discussion for September 2019

NORD Honors Industry In Patient Advocates At Rare Impact Awards Gala & Overprotective Parents

Anti-Epileptic Keppra Can Be A Candidate For SMA Treatment & Batcave For Personal Space

Zolgensma’s Arrival Adds Urgency to SMA Newborn Screening Efforts & Natalie Russo And Her Brother

Eurordis' Integrated Care Initiative For Rare Disease Patients & Cooperate With Physical Therapists

RNA Molecule miR-23a May Have Therapeutic Role in SMA & From College Writing Course To A Career

Zolgensma’s Price And Its 'Cure One, Cure Many' Potential & Concerts Accessibility

#40 - Interview With Kellie Cusack

High Levels of Alpha-COP Proteins Seems To Ease SMA Severity in Mouse Models & Spinraza For 2 Years

The "Rare Barometer” Is Helping Eurordis Shape EU Rare Disease Policies & SMA Community Diversity

Zolgensma For SMA Types 1, 2 And 3, More Options On The Way & Angela Titcombe

Ontario Patients Are Granted With Wider Access To Spinraza & Current Treatments, Brighter Futures

Family Says Zolgensma Saved All of Their Lives; Their Gene Therapy & Tyler Dukes

Zolgensma's Potential To Change The Whole Landscape of SMA & Melissa Milinovich's Story

#39 - Interview With Jason Bertsch

European SMA Advocates Question The EU’s Willingness to Reimburse Zolgensma & Independence

Improved Survival And Key Motor Milestones Achieved With Risdiplam & Stress Management

Results From Phase 2 Trial of Spinraza in Infants With SMA Support Efficacy And Safety & Sleeping

Spinraza Is Showing Long-Term Benefits In All SMA Types & SMA Awareness Month

AveXis States Zolgensma Is Showing Strong Efficacy Across SMA Types & Coping With Bad Days

Spinraza Continues to Demonstrate Safety and Improvements in SMA Patients & Tips For Giving Hugs

Will The National Institute For Health And Care Recommend Spinraza & Treating Each Day As A Blessing

Will NIH And NICE Recommend Funding of Spinraza in England? & Each Day As A Blessing