By 2025, between 10 and 20 new gene therapies could win approval annually by the US Food and Drug Administration (FDA), most of them delivered via adeno-associated virus (AAV). Yet Duchenne muscular dystrophy (DMD) presents some unique challenges for AAV-delivered gene therapy — namely because the dystrophin gene, which is missing in DMD, is one of…
Six biotech companies working toward a cure for Duchenne muscular dystrophy summarized their progress during an online conference session on exon skipping.
The session was an informal chat where the moderators and panelists talked about what it is like to live with a rare disease like DMD.
Rare Disease Advisor is excited to cover CureDuchenne’s 2021 FUTURES Nationals Conference. The CureDuchenne 2021 FUTURES National Conference is a virtual event focused on bringing education, resources, and connection to the Duchenne community in a family-friendly environment. This year’s conference agenda and special events will focus on Quality of Life. CureDuchenne will be offering multiple robust presentations and panel discussions, a Symposium, a Resource Fair, e-Gaming experiences, and more.
