Michael Kelly, MD, chief scientific officer at CureDuchenne, says a gene therapy for DMD could win FDA approval as early as 2023.
Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Michael Kelly, PhD, chief scientific officer at CureDuchenne, on treatments for DMD.
Thousands of families affected by Duchenne in the US and Latin America face the extra hurdle of accessing reliable information on the disorder in Spanish.
The nonprofit Team Joseph raises money for scientific studies, but also looks for immediate gaps or needs not being met by other patient organizations.
Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Marissa Penrod, founder of the Indiana-based nonprofit group Team Joseph.
A new-generation gene therapy to treat DMD was introduced at the CureDuchenne 2022 FUTURES National Conference.
The speakers at a CureDuchenne FUTURES conference session I took in were mothers of children and an adult with Duchenne muscular dystrophy (DMD).
The different types of gene therapy to treat Duchenne muscular dystrophy (DMD) were outlined at a symposium at the CureDuchenne 2022 FUTURES conference.
Three medical providers gave presentations on how to balance these factors with the lifestyles that surround Duchenne muscular dystrophy (DMD).
The session discussed the rationale behind the mechanisms of endogenous dystrophin restoration for the treatment of DMD.
The present and future of vamorolone were described at the CureDuchenne 2022 FUTURES National Conference by Santhera’s patient advocacy lead.
CureDuchenne’s national FUTURES conference on DMD will be held between May 27, 2022, and May 29, 2022, in Orlando, Florida, and virtually.