News

October 15, 2020October 15, 2020

Edasalonexent Helps to Preserve Heart and Bone Health, New Data Show

News

Edasalonexent, a potential oral therapy for Duchenne muscular dystrophy (DMD), can prevent the development of heart disease and help to preserve bone health, according to data from two studies ... Read more

October 13, 2020October 13, 2020

Phase 3 Trial of Puldysa Stopped for Lack of Efficacy in Duchenne Patients

News

Santhera Pharmaceuticals has decided to stop SIDEROS, its Phase 3 trial assessing the effectiveness of Puldysa (idebenone) at delaying respiratory decline in patients with Duchenne muscular dystrophy (DMD) on ... Read more

October 8, 2020October 8, 2020

Pfizer’s Gene Therapy for Duchenne Placed on Fast Track by FDA

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The U.S. Food and Drug Administration (FDA) has granted fast track designation to PF-06939926, Pfizer’s investigational gene therapy for Duchenne muscular dystrophy (DMD). This designation is given to treatments ... Read more

October 7, 2020October 7, 2020

Same But Different Asks Public to Vote on ‘Glimmer of Hope’ Calendar Photos

News, syndicated

Same But Different, a nonprofit U.K. group that uses art for social change, is inviting people to choose their favorite photographs in a calendar contest to heighten awareness of rare ... Read more

October 6, 2020October 6, 2020

FDA Lifts Clinical Hold on IGNITE DMD Gene Therapy Trial

News

The U.S. Food and Drug Administration (FDA) has lifted its clinical hold on the Phase 1/2 trial testing SGT-001, Solid Biosciences’ investigational gene therapy for people with Duchenne muscular ... Read more

October 1, 2020October 1, 2020

Durable Safety and Motor Gains Evident in Trial of SRP-9001, DMD Gene Therapy

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A single dose of SRP-9001, Sarepta Therapeutics’ investigational gene therapy for Duchenne muscular dystrophy (DMD), continues to be safe and to improve motor function in four boys treated two years ago, according ... Read more

September 29, 2020September 29, 2020

Givinostat Wins Rare Pediatric Disease Designation; Phase 3 DMD Trial Fully Enrolled

News

Italfarmaco has now completed recruitment — delayed in the spring due to the COVID-19 pandemic — of boys with Duchenne muscular dystrophy (DMD) for its ongoing Phase 3 trial of givinostat, ... Read more

September 28, 2020September 28, 2020

Efforts Continue Beyond Awareness Month to Expand Newborn Screening Panels in US

News, syndicated

It took one year for Dona Krystosek to get a diagnosis for her son, Levi, after he was born. The family received three misdiagnoses of fatal diseases until they ... Read more

September 24, 2020September 24, 2020

Vamorolone Continues to Improve Motor Function in DMD Boys, Phase 2 Trials Show

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The experimental therapy vamorolone can improve motor function significantly in boys with Duchenne muscular dystrophy (DMD), without stunting their growth and with fewer side effects compared to standard corticosteroids, according to 18-month ... Read more

September 22, 2020September 22, 2020

Muscular Dystrophy and Other Clinical Trials Adapting to Life With COVID-19

News

In a Facebook Live event hosted by the Muscular Dystrophy Association (MDA), experts discussed the effect that the COVID-19 pandemic has had on research efforts in muscular dystrophy, as ... Read more

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