Pat Furlong, BSN, RN, on the State of Gene Therapy in Muscular Dystrophy


The founding president and chief executive officer of Parent Project Muscular Dystrophy discussed recent and upcoming milestones in the field.

“There's no broadly applicable treatment at this moment to address dystrophin production. We have 4 approvals of antisense oligonucleotides that really only apply to around 20% of the population... The access issues are difficult for us... we've had some negative payer policies and those are likely to inhibit access for certain people, which isreally heartbreaking for all of us because we are very anxious for all of these boys to have treatment ensured.”

While 2023 has seen lots of progress in the treatment landscape of the field, including the summer approval of delandistrogenemoxeparvovec (marketed as Elevidys by Sarepta), the field’s first gene therapy, more work always remains to be done. September is Duchenne Action and Muscular Dystrophy Awareness Month, and CGTLive is speaking with clinicians and advocacy groups in the space to learn more about the progress in the field to date and unmet needs that remain.

CGTLive spoke with Pat Furlong, BSN, RN, founding president and chief executive officer, Parent Project Muscular Dystrophy (PPMD), to learn more about her thoughts on the progress to date in the field of muscular dystrophies. She lauded the first-of-its-kind FDA approval of Elevidys and touched on other disease-modifying therapies (DMTs) available for Duchenne Muscular dystrophy (DMD) but stressed that most of the patient population of DMD still lack DMTs, and even for patients indicated for DMTs, many factors get in the way of actually accessing these therapies. She also shared milestones she is looking forward to in the field, such as PDUFA dates for other modes of therapy, including vamorolone and givinostat.

Sarepta Therapeutics announces FDA approval of ELEVIDYS, the first gene therapy to treat Duchenne muscular dystrophy. News release. Sarepta Therapeutics. June 22, 2023.
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