Natalie Goedeker, CPNP, on Supporting Gene Therapy’s Ascendance in Muscular Dystrophies

"Duchenne muscular dystrophy was denied being added to the recommended newborn screening panel. ...the community has to focus on getting data [in this disease] in the coming years. Probably it will require some sort of clinical trial, but we definitely need data in young children showing that it makes a difference, treating them that their diagnosis from birth will make a difference versus a symptomatic diagnosis in outcomes once we do have a good treatment.”

One track, chaired by Natalie Goedeker, CPNP, pediatric neurology, at the 2023 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, March 19-22, in Dallas, Texas was the Practical Considerations in Gene Therapy Track that discussed unmet research, education, and infrastructure needs that must be addressed across rare dystrophies such as spinal muscular atrophy and Duchenne muscular dystrophy (DMD).

CGTLive spoke with Goedeker to learn more about the conversations that arose during the practical considerations in gene therapy track. She discussed the importance of having rare diseases like DMD on newborn screening panels and data that will need to come out in the near future to support that implementation. She also touched on positive patient and physician perspectives on accelerated approval shared during one of the sessions on the track at the conference.

Read more coverage of the 2023 MDA Conference here.

REFERENCE

Practical Considerations in Gene Therapy Track. Presented at: 2023 MDA Conference, March 19-22; Dallas, Texas.