New Drug For Duchenne Muscular Dystrophy With Dr. Steven Quay

Send us Fan MailIn this episode of Rare Connection, I’m joined by physician-scientist and biotech CEO Dr. Steven Quay to discuss an emerging approach to treating Duchenne muscular dystrophy—and how innovation in one area of medicine may open doors for others.Dr. Quay is the CEO of Atossa Therapeutics, where his team is developing (Z)-endoxifen, a next-generation endocrine therapy originally studied in breast cancer. Unlike tamoxifen, which must be metabolized in the body, (Z)-endoxifen delivers the active compound directly—helping to reduce variability between patients and improve consistency in dosing and response.What makes this conversation especially important for the rare disease community is the potential application of this therapy in Duchenne muscular dystrophy. Research suggests that (Z)-endoxifen may help increase levels of utrophin, a protein that could compensate for the lack of dystrophin in individuals living with Duchenne.We also discuss:What makes Duchenne muscular dystrophy a target for this type of therapyThe significance of Orphan Drug DesignationHow drugs developed for one condition may have applications in othersThe challenges of bringing new treatments from research to patientsDuchenne muscular dystrophy affects approximately 1 in 3,500 to 5,000 boys worldwide, and while progress is being made, many families are still waiting for effective, accessible treatments.👉 Coming Next: During this conversation, Dr. Quay also introduced the potential application of (Z)-endoxifen in McCune-Albright syndrome, an ultra-rare condition. We’ll be continuing this discussion in a follow-up episode focused specifically on that topic.This episode offers insight into how innovation in drug development may create new possibilities—not only for Duchenne, but for the broader rare disease community.🔗 Support the ShowIf you’d like to support Rare Connection, you can use the “Support the Show” link in the show notes. Contributions help continue conversations like this and expand awareness for rare diseases.💬 Connect & ShareFollow Rare Connection on Facebook, X, and LinkedInWatch full episodes on YouTube at Rare ChefUse the “Send Me a Text” link in the show notes to share your thoughts or questions⚠️ DisclaimerThis podcast is for informational and educational purposes only and is not intended as medical advice. Always consult with a qualified healthcare professional regarding any medical condition or treatment.Support the show