Muscular Dystrophy News

PTC Therapeutics plans to soon ask the FDA to approve ataluren to treat Duchenne muscular dystrophy (DMD) caused by nonsense mutations. https://buff.ly/3zE2wGA

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Columnist Betty Vertin, already a full-time mother and caregiver, goes back to work full time to meet the financial burdens of DMD care. https://buff.ly/3YOpx4i

Del-zota, formerly AOC 1044, increased levels of dystrophin production to 25% of normal in DMD patients in the EXPLORE44 trial, per new data. https://buff.ly/3yRq9eK

While things have been difficult lately for columnist Shalom Lim, who is at a crossroads in life, out of suffering comes redemption. https://buff.ly/3M5IhVy

How have Parent Project Muscular Dystrophy and its annual conference contributed to the muscular dystrophy community over the past 30 years? Trace the achievements here: https://buff.ly/4cDUCfe

FSHD affects one's identity. For columnist Robin Stemple, that means redefining what it means to be a man. https://buff.ly/3Ax75Tw

Up and coming treatments, including approaches in gene therapy and regenerating muscle cells, were highlighted at the PPMD conference. Learn more here. https://buff.ly/4d2EyDm

The FDA has granted rare pediatric disease designation to SAT-3247, Satellos' oral therapy candidate for Duchenne muscular dystrophy (DMD). https://buff.ly/3WMo2AS

Connecting with a supportive community is essential for maintaining both emotional and physical well-being for people with muscular dystrophy. Learn more: https://buff.ly/3RX8c4P

Shalom Lim recalls his special relationship with his caregiver of 17 years and touches on the lasting power of love and support. https://buff.ly/4dBBz54

Learn more about the largest global gathering of Duchenne and Becker families and how the 30th annual conference aimed to help families in attendance: https://buff.ly/4cG6v47

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More than a half dozen exon-skipping therapies for DMD, all aiming to increase dystrophin protein production, are being explored in trials. Learn more: https://buff.ly/4cqt399

Columnist Betty Vertin loved seeing her daughter's friends include her sons with Duchenne on the wedding dance floor. https://buff.ly/4cAkyaR

The experimental gene therapy RGX-202 has been well tolerated and is showing signs of efficacy in boys with DMD in a Phase 1/2 trial, data show. https://buff.ly/3ylQ0eR

Robin Stemple reflects on his many sources of support and the ways they continue to shape his journey with FSHD and blindness. https://buff.ly/3YFBKIr

Columnist Betty Vertin says that when her sons suffer from treatment, she sometimes questions what she's doing. There are no easy answers. https://buff.ly/4dtmse2

Clinical trials testing gene therapies for DMD were a topic of discussion at last week's Parent Project Muscular Dystrophy conference. https://buff.ly/3VWpArM

While things have been difficult lately for columnist Shalom Lim, who is at a crossroads in life, out of suffering comes redemption. https://buff.ly/3ywfne0

Safety was seen in 3 Duchenne MD patients given PGN-ED051 at low dose in ongoing Phase 2 study and greater to dystrophin protein production. https://buff.ly/4dan0FL

Pfizer discontinued development of its investigational gene therapy for DMD after the treatment failed to meet Phase 3 trial goals. https://buff.ly/3SBqzN8

Retired music teacher Patrick Moeschen continues his advocacy work and writing in a new column about his life with LGMD. https://buff.ly/4dxOB3t

Transition planning is a continuous process, but the groundwork can start in the early teen years. Here’s what you should know: https://buff.ly/3Uuhwhi

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The last few days have been spectacular for the Vertin family. Columnist Betty Vertin shares how finding beauty is also part of Duchenne. https://buff.ly/3SybEmM

Starting school is an exciting milestone. Don’t let DMD overshadow it for your family. Here’s how you can prepare: https://buff.ly/4dirxGc

Pfizer discontinued development of its investigational gene therapy for DMD after the treatment failed to meet Phase 3 trial goals. https://buff.ly/3A8Z5rV

Columnist Betty Vertin wants fields of dreams and hopes for all her children — including the one who wants to play college baseball. https://buff.ly/3SyQULU