WDO Webinar: Gene Therapy in Duchenne Muscular Dystrophy

The WDO webinar on 'Gene Therapy in Duchenne Muscular Dystrophy,' held on November 7, 2023, delved into crucial aspects of gene therapy for DMD patients.
Topics:
3:11 How gene therapy works - Prof Annemieke Aartsma-Rus
30:43 Managing serious adverse events (SAE) - Prof Eugenio Mercuri
50:38 Immune responses in gene therapy - Prof Francesco Muntoni
1:10:59 Gene therapy and the heart - Dr Chet Villa, MD
1:46:16 Gene therapy landscape and clinical trials - Company presentations
2:30:38 Access and reimbursement: The SMA experience - Mencía de Lemus Belmonte
© 2023 World Duchenne Organization.
Medical disclaimer: Nothing posted on the channel is medical advice or a substitute for advice from your physician or healthcare provider. Always contact your physician or other healthcare provider with any questions about a medical condition or your personal health.

[@rajuyadavyadav9419]

Please help me for DMD disease

[@blessedservant]

It is sad that older ambulatory kiddos without the AAV antibodies are not given an exception, and allowed to participate in the trial for microdystrophin. Time is muscle. Please give these boys a chance at life.