These Kids Can't Wait

Episode 12: An Update from The Lost Enzyme ProjectThe founders of The Lost Enzyme Project share the journey to develop a treatment for Beta-Mannosidosis—how far we’ve come and how far we still have to go.From early momentum to real progress, this episode offers a behind-the-scenes look at what it takes to move a rare disease treatment forward—the challenges, the milestones, and the urgency driving it all. Because for our kids, waiting isn’t an option.🎧 Listen nowLearn more about The Lost Enzyme Project:🔗 https://tlep.org (https://tlep.org/)Support the mission and follow along:🔗 https://thelostenzymeproject.org/podcast/

🎙️ Hosts: Erin Hubbard🎤 Guest: Nikki Mcintosh📅 Release Date: 11/17/2025🔬 Topic: RARE MAMAS-Empowering Strategies for Navigating Your Child’s Rare DiseaseEpisode OverviewIn this episode of These Kids Can’t Wait, host Erin Hubbard speaks with Nikki McIntosh, author of Rare Mamas: Empowering Strategies for Navigating Your Child’s Rare Disease and creator of the Rare Mamas Rising podcast. Nikki shares the mission behind her work. Providing resources, connection, and emotional support for parents raising medically complex or rare disease children. She reflects on the tools and mindsets she wished she had earlier in her journey and discusses how community, storytelling, and lived experience shaped her advocacy. Nikki also opens up about her life as a “rare mama” to her son with spinal muscular atrophy (SMA) and how that path inspired her to help others find clarity, connection, and strength.Key Takeaways✔️ The purpose behind the Rare Mamas Rising podcast and guidebook✔️ How storytelling and shared experiences reduce isolation in the rare disease community✔️ The evolving landscape of SMA care and treatment✔️ The emotional and practical challenges of navigating complex care systems✔️ Nikki’s mission to empower parents through education, mindset, and communityResources & LinksRaremamas.comhttps://www.instagram.com/rare_mamas/🌐 Learn more: thelostenzymeproject.org📢 Follow us on social media:https://www.linkedin.com/company/the-lost-enzyme-projecthttps://www.instagram.com/thelostenzymeproject/https://www.facebook.com/people/The-Lost-Enzyme-Project/61551541563692/📖Books Mentioned in the Episode:Rare Mamas: Empowering Strategies for Navigating Your Child’s Rare Disease.raremamas.com/book📩 Contact us: Email us at [email protected] Producer: [email protected] & Stay ConnectedStay informed by subscribing to These Kids Can’t Wait on Spotify, Apple Podcasts, Youtube or Amazon music. If you find this episode valuable, please leave a review to help raise awareness for rare disease research.

Episode 10 – Finding Strength in Community🎙️ Hosts: Erin Hubbard🎤 Guest: Cyndi Frank📅 Release Date: 10/07/2025🔬 Topic: Resilience, Advocacy, and Rare Disease Awareness🎧 Episode OverviewIn this episode of These Kids Can’t Wait, host Erin Hubbard speaks with Cyndi Frank, a leading advocate in the Gaucher disease community. In recognition of Gaucher Disease Awareness Month, Cyndi shares her journey of raising awareness, connecting advocates, and building strong partnerships with pharmaceutical companies. Together, they discuss how collaboration, transparency, and communication between patients, advocates, and industry can drive meaningful progress in rare disease research and treatment.Key Takeaways✔️ The significance of Gaucher Disease Awareness Month✔️ How advocates and industry can work together for better outcomes✔️ The importance of trust and communication in rare disease partnerships✔️ Challenges and opportunities in building sustainable collaborationResources & LinksGaucher Community Alliance: https://www.gauchercommunity.org/🌐 Learn more: thelostenzymeproject.org📢 Follow us on social media:https://www.linkedin.com/company/the-lost-enzyme-projecthttps://www.instagram.com/thelostenzymeproject/https://www.facebook.com/people/The-Lost-Enzyme-Project/61551541563692/📩 Contact us:Email us at [email protected]: [email protected] & Stay ConnectedStay informed by subscribing to These Kids Can’t Wait on Spotify, Apple Podcasts, Youtube or Amazon music. If you find this episode valuable, please leave a review to help raise awareness for rare disease research.In this episode, I accidentally mispronounced Gaucher disease as Gaucher's a few times near the beginning. My apologies for the mistake—thank you for understanding!

Episode 9: Turning Hope into Action🎙️ Hosts: Erin Hubbard and Laurel Gregier🎤 Guest: Kristin Andrus📅 Release Date: 9/12/2025🔬 Topic: Philanthropy, Family Values, and Building Stronger CommunitiesEpisode OverviewIn this episode of These Kids Can’t Wait, we sit down with Kristin Andrus—founder of Gathering for Impact, Sister Goods, and head of the Andrus Family Foundation. A passionate advocate for families, women, and vulnerable communities across Utah, Kristin shares her journey from growing up in a service-oriented home to creating platforms that mobilize thousands toward meaningful action.With honesty and warmth, she reflects on lessons learned from refugee mentorship, raising six children in a culture of giving, and why real impact often starts at the local level. Together, we explore how philanthropy, social media, and grassroots service intersect to create lasting change.Key Takeaways✔️ How family values and upbringing shape a life of service✔️ The importance of “getting your hands dirty and your heart a little broken”✔️ Building a culture of giving in your family and community✔️ Practical advice for nonprofits with small but passionate bases of support✔️ Why authentic connection matters more than numbers in fundraising and advocacyResources & Links🌐Learn more: thelostenzymeproject.org📢 Follow us on social media:https://www.linkedin.com/company/the-lost-enzyme-projecthttps://www.instagram.com/thelostenzymeproject/https://www.facebook.com/people/The-Lost-Enzyme-Project/61551541563692/Kristin's Website: https://kristinandrus.com/Gathering for Impact: https://www.gatheringforimpact.org/Giving personality test: https://byu.az1.qualtrics.com/jfe/form/SV_1NTleJAzYPR868K📩 Contact us:Email us at [email protected]: [email protected] & Stay ConnectedStay informed by subscribing to These Kids Can’t Wait on Spotify, Apple Podcasts, Youtube or Amazon music. If you find this episode valuable, please leave a review to help raise awareness for rare disease research.

🎙️ Hosts: Erin Hubbard, Laurel Gregier, and Lorena Lomelin🎤 Guest: Mathias Schmidt📅 Release Date: 7/11/2025🔬 Topic: Breaking the Blood-Brain Barrier, Compassionate Use, and the Power of Patient-Centered InnovationEpisode OverviewIn this episode of These Kids Can’t Wait, we’re joined by Mathias Schmidt, President and CEO of Armagen and Executive Fellow at JCR Pharmaceuticals. With over 20 years in rare disease research, Dr. Schmidt discusses how science and empathy intersect in the development of treatments for lysosomal storage diseases—particularly those affecting the brain. From pioneering enzyme therapies that cross the blood-brain barrier to founding The Lost Enzyme Project, Mathias offers a hopeful, grounded vision for what’s possible when patients are at the center of innovation.Key Takeaways✔️ How JCR’s J-Brain Cargo technology delivers enzymes past the blood-brain barrier✔️ Why focusing on quality of life—rather than a cure—is a powerful shift in mindset✔️ Reflections on the emotional and ethical dimensions of working in rare diseaseResources & Links🌐 Learn more: thelostenzymeproject.org📢 Follow us on social media:https://www.linkedin.com/company/the-lost-enzyme-projecthttps://www.instagram.com/thelostenzymeproject/https://www.facebook.com/people/The-Lost-Enzyme-Project/61551541563692/📩 Contact us:Email us at [email protected]: [email protected] & Stay ConnectedStay informed by subscribing to These Kids Can’t Wait on Spotify, Apple Podcasts, Youtube or Amazon music. If you find this episode valuable, please leave a review to help raise awareness for rare disease research.

🎙️ Hosts: Erin Hubbard 🎤 Guest: Dr. Candice Wood 📅 Release Date: 6/9/2025 🔬 Topic: Postpartum Mental Health, Holistic Women’s Care, Motherhood & Burnout, Supporting Rare Disease Families Episode OverviewIn this episode, we sit down with Dr. Candice Wood, OB-GYN and founder of Women Made Well, a practice dedicated to supporting women through both physical and emotional transitions. Dr. Wood shares how her experiences as a physician and a mother inspired her to redefine what true healing looks like—one that embraces emotional wellbeing as deeply interconnected with physical health.Key Takeaways✔️How traditional OB-GYN care often falls short on mental health✔️Why Dr. Wood founded Women Made Well✔️The realities of postpartum anxiety and depression✔️How social media exacerbates feelings of inadequacy in new mothers✔️Practical strategies for navigating motherhood with a growth mindset✔️The importance of community, even when living with rare diseases✔️When and how medical intervention fits into emotional healing Resources & Links🌐 Learn more: thelostenzymeproject.orgWomen Made Well Podcast: https://www.womanmadewell.com/woman-made-well-podcast📢 Follow us on social media:https://www.linkedin.com/company/the-lost-enzyme-projecthttps://www.instagram.com/thelostenzymeproject/https://www.facebook.com/people/The-Lost-Enzyme-Project/61551541563692/📩 Contact us:Email us at [email protected]: [email protected]📖Books Mentioned in the Episode (ISBN):147213995X14019713691622039076Subscribe & Stay ConnectedStay informed by subscribing to These Kids Can’t Wait on Spotify, Apple Podcasts, Youtube or Amazon music. If you find this episode valuable, please leave a review to help raise awareness for rare disease research.

🎙️ Hosts: Erin Hubbard and Laurel Gregier 🎤 Guest: Dr. Paul Orchard 📅 Release Date: 5/12/2025 🔬 Topic: Bone Marrow Transplants, Gene Therapy, and the Future of Rare Disease Treatment Episode OverviewIn this episode of These Kids Can’t Wait, we are joined by Dr. Paul Orchard, Medical Director of the Inherited Metabolic and Storage Disease Transplantation Program at the University of Minnesota. Dr. Orchard brings decades of experience treating ultra-rare lysosomal disorders through stem cell transplantation and emerging gene therapies. Together with hosts Erin and Laurel, he walks us through the science, challenges, and hope behind bone marrow transplants for rare diseases, sharing insights from his career, reflections on patient care, and the future of life-saving treatment. Key Takeaways✔️ How bone marrow transplantation works for lysosomal storage disorders ✔️ The difference between cord blood and unrelated donor stem cells ✔️ The impact of early diagnosis and newborn screening ✔️ Limitations of enzyme replacement therapy and the promise of gene therapy ✔️ How families and advocates can help shape research and access to treatment ✔️ The emotional and medical realities of caring for ultra-rare patients Resources & Links🌐 Learn more: thelostenzymeproject.org📢 Follow us on social media:https://www.linkedin.com/feed/https://www.instagram.com/thelostenzymeproject/https://www.facebook.com/people/The-Lost-Enzyme-Project/61551541563692/📩 Contact us:Email us at [email protected]: [email protected] Subscribe & Stay ConnectedStay informed by subscribing to These Kids Can’t Wait on Spotify, Apple Podcasts, Youtube or Amazon music. If you find this episode valuable, please leave a review to help raise awareness for rare disease research.

Episode 5: A Deep Dive into Rare Disease Research: Part 2🎙️ Hosts: Erin Hubbard, Laurel Gregier🎤 Guest: Dr. Martin📅 Release Date: 4/21/2025🔬 Topic: Advancing Research for Beta-MannosidosisEpisode OverviewIn Part 2 of our conversation with Dr. Angela Martin, we are continuing our conversation on rare disease research. Building on our earlier discussion of enzyme replacement therapy and the challenges of treating beta-mannosidosis, this episode explores how awareness, advocacy, and day-to-day lab work come together to drive progress.Dr. Martin shares her passion for science, the emotional impact of working closely with families, and how conferences like WORLD play a pivotal role in forging critical partnerships. We also talk about the real-life stories and responsibilities behind the science, and what keeps researchers like Dr. Martin going in the face of uncertainty.Key Takeaways✔️ The importance of awareness and early diagnosis in rare disease treatment✔️ How parent-led advocacy accelerates scientific progress✔️ The daily grind—and joy—of conducting rare disease research✔️ The pivotal role of conferences like WORLD in connecting research teams with potential biotech partners✔️ How families and supporters can meaningfully contribute to ongoing researchResources & Links🌐 Learn more: thelostenzymeproject.org📢 Follow us on social media:https://www.linkedin.com/feed/https://www.instagram.com/thelostenzymeproject/https://www.facebook.com/people/The-Lost-Enzyme-Project/61551541563692/📩 Contact us:Email us at [email protected]: [email protected] & Stay ConnectedStay informed by subscribing to These Kids Can’t Wait on Spotify, Apple Podcasts, Youtube or Amazon music. If you find this episode valuable, please leave a review to help raise awareness for rare disease research.

🎙️ Hosts: Erin Hubbard and Laurel Gregier🎤 Guest: Dr. Angela Martin📅 Release Date: 4/8/2025🔬 Topic: Understanding Beta-Mannosidosis and the Innovation Behind Enzyme Replacement TherapyEpisode OverviewIn this episode of These Kids Can’t Wait, hosts Erin Hubbard and Laurel Gregier are joined by Dr. Angela Martin, a postdoctoral researcher at the Kimonis Laboratory and a vital force in The Lost Enzyme Project. Together, we discuss beta-mannosidosis, a rare lysosomal storage disorder, and the innovative efforts underway to develop treatments, including a promising new therapy using J-Brain Cargo technology.Key Takeaways✔️ What beta-mannosidosis is and why it's so difficult to diagnose and treat✔️ How J-Brain Cargo technology enables enzyme delivery across the blood-brain barrier✔️ The stages of drug development—from lab to clinical trials✔️ Why underdiagnosis is a major issue for ultra-rare diseases✔️ The power of international collaboration in rare disease innovationResources & Links🌐 Learn more: thelostenzymeproject.org📢 Follow us on social media:https://www.linkedin.com/feed/https://www.instagram.com/thelostenzymeproject/https://www.facebook.com/people/The-Lost-Enzyme-Project/61551541563692/📩 Contact us:Email us at [email protected]: [email protected] & Stay ConnectedStay informed by subscribing to These Kids Can’t Wait on Spotify, Apple Podcasts, Youtube or Amazon music. If you find this episode valuable, please leave a review to help raise awareness for rare disease research.

🎙️ Host: Erin Hubbard🎤 Guest: Sarah Forsman📅 Release Date: 2/28/2025🔬 Topic: Life with Alpha-Mannosidosis and the Impact of Early TreatmentEpisode OverviewIn this episode of These Kids Can’t Wait, host Erin Hubbard speaks with Sarah Forsman, a patient living with Alpha-Mannosidosis. Sarah shares her personal journey, including undergoing a bone marrow transplant at the age of four, and how this early intervention has shaped her life.From navigating the challenges of a rare disease to advocating for greater awareness, Sarah offers a unique and inspiring perspective on what it means to live with Alpha-Mannosidosis. This conversation sheds light on the realities of managing a lysosomal storage disorder, the importance of early diagnosis and treatment, and the resilience of the rare disease community.Key Takeaways✔️ Sarah’s experience with Alpha-Mannosidosis and her early treatment journey✔️ The impact of a bone marrow transplant on disease progression✔️ Challenges and triumphs of living with a rare disease✔️ The importance of research, advocacy, and patient support networksResources & Links🌐 Learn more about The Lost Enzyme Project: https://thelostenzymeproject.org/ 📢 Follow us on social media: https://www.linkedin.com/feed/https://www.instagram.com/thelostenzymeproject/https://www.facebook.com/people/The-Lost-Enzyme-Project/61551541563692/📩 Contact us: Email us at [email protected] to get in contact with our producer at [email protected]’s Blog: https://achievetheimpossibletoday.com/blog/ISMRD: https://www.ismrd.org/Subscribe & Stay ConnectedStay informed by subscribing to These Kids Can’t Wait on Spotify, Apple Podcasts, Youtube or Amazon music. If you find this episode valuable, please leave a review to help raise awareness for rare disease research.

🎙️ Hosts: Lorena Lomelin and Phoebe Wang 🎤 Guest: Dr. Virginia Kimonis 📅 Release Date:2/28/2025 🔬 Topic: A deep dive into rare disease research.Episode Summary:In this episode of These Kids Can’t Wait, hosts Lorena Lomelin and Phoebe Wang sits down with Dr. Virginia Kimonis, a nationally renowned expert in genetics and clinical biochemical genetics, to discuss her groundbreaking work on Beta-Mannosidosis and the development of a novel treatment. Dr. Kimonis shares her journey into rare disease research, the challenges of treating lysosomal storage disorders, and the innovative science behind The Lost Enzyme Project. We also explore the impact of this research on patients and families, the funding challenges in rare disease research, and the future of treatments for ultra-rare conditions.Key Takeaways:✔️ What lysosomal storage disorders are and why they’re challenging to treat ✔️ An overview of Beta-Mannosidosis, its symptoms and the challenges faced ✔️ The process of developing a treatment, from preclinical research to clinical trials ✔️ The future of rare disease research and the potential for gene therapyResources & Links🌐 Learn more: https://thelostenzymeproject.org/ or https://bit.ly/lostenzyme 📢 Follow us on social media: https://www.linkedin.com/feed/ https://www.instagram.com/thelostenzymeproject/ https://www.facebook.com/people/The-Lost-Enzyme-Project/61551541563692/ 📩 Contact us: Email us at [email protected] to get in contact with our producer at [email protected] Dr. Kimonis’s lab: https://www.mammag.uci.edu/kimonis/index.asp Subscribe & Stay ConnectedStay informed by subscribing to These Kids Can’t Wait on [Spotify, Apple Podcasts, or preferred platform]. If you find this episode valuable, please leave a review to help raise awareness for rare disease research.

🎙️ Hosts: Erin Hubbard, Laurel Gregier, and Lorena Lomelin📅 Release Date:2/28/2025🔬 Topic: Story Behind the Lost Enzyme ProjectEpisode OverviewIn this inaugural episode of These Kids Can’t Wait, we are joined by the co-founders of The Lost Enzyme Project—Erin Hubbard, Laurel Gregier, and Lorena Lomelin. As parents of children affected by ultra-rare lysosomal storage disorders, they share their deeply personal experiences, the challenges of advocating for research in rare diseases, and the motivation behind launching this initiative.This discussion highlights the urgent need for scientific progress, the barriers to treatment development, and the power of patient-led advocacy in accelerating change.Key Takeaways✔️ The personal journeys that led to the creation of The Lost Enzyme Project✔️ The organization’s mission to drive research and innovation for rare diseases✔️ Challenges in the rare disease space, including funding gaps and limited awareness✔️ The importance of collaboration between families, researchers, and biotech leadersResources & Links🌐 Learn more: https://thelostenzymeproject.org/📢 Follow us on social media: https://www.linkedin.com/feed/https://www.instagram.com/thelostenzymeproject/https://www.facebook.com/people/The-Lost-Enzyme-Project/61551541563692/📩 Contact us: Email us at [email protected] to get in contact with our producer at [email protected] & Stay ConnectedStay informed by subscribing to These Kids Can’t Wait on Spotify, Apple Podcasts, Youtube or Amazon music. If you find this episode valuable, please leave a review to help raise awareness for rare disease research.