It’s been nearly two years since Elevidys (delandistrogene moxeparvovec-rokl), a gene therapy for Duchenne muscular dystrophy (DMD) developed by Sarepta Therapeutics, was approved by the U.S. Food and Drug Administration (FDA), marking the first time a gene therapy for DMD was authorized in the U.S. Since its…
I recently penned a column that took a surface look at the history of the disability movement in the United States. As many readers pointed out, my list of world changers and their accomplishments was far from exhaustive. I’d like to share another small part of the movement that…
The U.S. Food and Drug Administration (FDA) has cleared Sarepta Therapeutics to start dosing and screening in a first clinical trial testing the gene therapy SRP-9005 in people with limb-girdle muscular dystrophy type 2C (LGMD2C). Sarepta said advancement continues for SRP-9003 and SRP-9004, two gene therapies…
I have friends and family who seem to be living on a medical merry-go-round. Every day they go to doctor’s appointments, have medical tests, or go through therapy. Their lives revolve around their healthcare. I haven’t been on this type of merry-go-round for a long time. I had a…
The Japan Ministry of Health, Labour and Welfare (MHLW) has granted orphan drug status to delpacibart etedesiran, known as del-desiran, an investigational therapy from Avidity Biosciences now in clinical testing for myotonic dystrophy type 1 (DM1). This designation is intended to support the development of therapies for rare…
Health Canada has agreed to review an application by Kye Pharmaceuticals seeking the approval of vamorolone for treating Duchenne muscular dystrophy (DMD) — and granted it priority review. Priority review shortens the review period from the standard 10 months to six. It’s given to therapies that have the…
I recently had the opportunity to attend a Singaporean theater production titled “Supervision,” thanks to the SingHealth Patient Advocacy Network (SPAN). SPAN is a collective of patients and caregivers who provide feedback to improve healthcare here in Singapore. I’ve been part of it since 2022. The invitation to the play…
An independent data monitoring committee (DMC) favors continuing dosing Elevidys (delandistrogene moxeparvovec-rokl) to people with Duchenne muscular dystrophy (DMD) in ongoing clinical trials, according to developer Sarepta Therapeutics. The determination comes after recruitment and dosing in certain clinical trials were paused after a young man who…
This August will mark 40 years since I was diagnosed with what we eventually discovered was limb-girdle muscular dystrophy. Though it’s taken me many years to accept my condition, it’s progressed slowly over the past four decades — and in that way, it’s somewhat akin to the…
I don’t gamble. I don’t know how to do it. I wouldn’t even know how to place a bet, and the only gambling game I recognize by name is poker. However, for someone who’s never gambled, I sure understand what it must feel like. How? Because I’m the caregiver of…
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