-
AB-1003 trial for LGMD proceeds after positive recommendation
Asklepios Biopharmaceutical (Askbio) has dosed the first participant in the second group of a Phase 1/2 trial testing AB-1003, an experimental gene therapy for limb-girdle muscular dystrophy (LGMD) type 2I/R9. The Phase 1/2 LION-CS101 trial (NCT05230459) is assessing AB-1003’s safety and tolerability in adults with LGMD2I/R9. Enrollment of the second group, which is ongoing at…
-
Advocates Unite in Washington D.C. to Champion Duchenne Policy Reforms
PPMD, with a group of more than 150 advocates, heads to Capitol Hill today to meet with Congressional leaders during this year’s annual PPMD Advocacy Conference. This marks the 23rd year that PPMD advocates will gather in Washington to urge Congress to advance legislation that will bring us closer to ending Duchenne. “Our community has…
-
Living with muscular dystrophy, from the perspective of 4 decades
A close friend of 30 years recently asked what my life was like before I was diagnosed with limb-girdle muscular dystrophy. When I met this man, we were both in college, studying music and playing drums. It was 1991, and I’d been diagnosed with muscular dystrophy for only six years. (Originally, doctors said I had…
-
In Case You Missed It…
Quest Media is an innovative adaptive lifestyle platform from MDA. With the power of this platform, we foster awareness and empowerment and have important conversations with experts, thought leaders, and members of the neuromuscular disease community about topics that matter to them and to the larger community of individuals with disabilities. With so many valuable…
-
Scientists unlock clues to new treatments for muscular dystrophy
Researchers combined advanced imaging techniques and theoretical physics to observe and explain how nanoclusters of the protein emerin form inside living cells. The study uncovers the molecular ‘rules’ driving the arrangement of emerin into nanoclusters and the mechanisms leading to their defective assembly in people with muscular dystrophy.
-
Quest Podcast: PJ’s Protocol: A Lifesaving Procedure Fueled by Love
In this episode of Quest Podcast, we chat with three pillars of the Duchenne muscular dystrophy community who are here with us on the 10th anniversary of PJ’s protocol. First, we have Brian Nicholoff whose son’s untimely passing was the catalyst for the creation of PJ’s protocol. Next is Amy Aikens whose son’s life was…
-
FDA grants priority review to deramiocel for DMD heart disease
The U.S. Food and Drug Administration (FDA) has accepted Capricor Therapeutics’ application seeking approval of deramiocel — a cell therapy developed by the biotech company to treat heart muscle disease in people with Duchenne muscular dystrophy (DMD) — and granted it priority review that will speed the agency’s decision. Priority review, which shortens the review…
-
As I reflect on life with Duchenne MD, I feel vindicated
Two weeks ago, I stood on stage at the Fullerton Hotel Singapore to receive a Study U.K. Alumni Award for “exceptional efforts in driving inclusion and positive change in society.” The British high commissioner to Singapore handed me the award while my partner, Amanda, and my caregiver, Glenda, cheered me on. It was a bittersweet…
-
Capricor Therapeutics Announces FDA Acceptance and Priority Review of Biologics License Application for Deramiocel
PPMD is excited to learn that the FDA has accepted Capricor Therapeutics’ Biologics License Application (BLA) for review, seeking traditional approval for deramiocel (CAP-1002). Additionally, the FDA granted the BLA Priority Review, reducing the review timeline from the standard 10-month to a priority six-month review from the date the submission is accepted by the FDA.…
-
MDA Ambassador Guest Blog: Finding My Way to a Sober and Healthy Way of Living
Cassidy Nilles is 35 years old and lives in Illinois. Cassidy lives with Limb Girdle muscular dystrophy (LGMD) type 2J. She lives with her daughter, Capri, and their maltipoo, Mila, in a ranch-style home that they share with her parents. After spending 10 years as a cosmetologist doing hair in both Los Angeles and Illinois,…
