Muscular Dystrophy News

August 13, 2020August 13, 2020

FDA Approves Viltolarsen, Now Viltepso, for DMD Patients With Exon 53 Mutation

The U.S. Food and Drug Administration (FDA) has conditionally approved NS Pharma‘s Viltepso (viltolarsen) for people with Duchenne muscular dystrophy (DMD) amenable to exon 53 skipping. Viltepso is given via ... Read more

August 11, 2020August 11, 2020

Patient Feedback Being Sought on Puldysa for Possible NHS Funding

Muscular Dystrophy News

The National Institute for Health and Care Excellence (NICE) is urging patient organizations to gather feedback from those with Duchenne muscular dystrophy (DMD) who received Santhera Pharmaceuticals‘ investigational treatment Puldysa ... Read more

August 6, 2020August 7, 2020

MD Advocates Offer Advice for Students With Neuromuscular Disorders Amid COVID-19

COVID-19 News, Muscular Dystrophy News

The importance of communication and self-care for people with muscular dystrophy and other neuromuscular diseases who are pursuing their education during the COVID-19 pandemic was highlighted in a Facebook Live ... Read more

August 4, 2020August 4, 2020

FDA Requests More Data, Maintains Clinical Hold on IGNITE DMD Trial of SGT-001 Gene Therapy

Muscular Dystrophy News

The U.S. Food and Drug Administration (FDA) is maintaining the clinical hold on the Phase 1/2 trial testing Solid Biosciences’ gene therapy SGT-001 for people with Duchenne muscular dystrophy (DMD), the company announced. ... Read more

July 30, 2020July 30, 2020

FDA Places SRP-9001, DMD Micro-dystrophin Gene Therapy, on Fast Track

Muscular Dystrophy News

An investigational gene therapy for Duchenne muscular dystrophy (DMD), called SRP-9001 micro-dystrophin, was given fast track designation by the U.S. Food and Drug Administration (FDA) its developer, Sarepta Therapeutics, announced in a ... Read more

July 28, 2020July 28, 2020

Stem Cell Therapy VTA-110 Named FDA Orphan Drug for DMD Treatment

Muscular Dystrophy News

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Vita Therapeutics‘ VTA-110, a candidate therapy for people with Duchenne muscular dystrophy (DMD). The designation grants various ... Read more

July 23, 2020July 23, 2020

Benitec Begins Early Study of BB-301 Gene Therapy for OPMD

Muscular Dystrophy News

Benitec Biopharma has begun the first study to support the start of clinical trials of BB-301, a potential gene therapy for people with oculopharyngeal muscular dystrophy (OPMD). The BB-301 Tissue Transduction Study ... Read more

July 21, 2020July 21, 2020

Two Imaging Biomarkers Predict Walking Ability in DMD, Useful for Trials

Muscular Dystrophy News

Two magnetic resonance measures — MRI and magnetic resonance spectroscopy (MRS) — of leg muscles are quality biomarkers that help to predict clinical milestones in Duchenne muscular dystrophy (DMD), including loss ... Read more

July 16, 2020July 16, 2020

NS Pharma Opens Support Hub to Help DMD Patients Access Treatments

Muscular Dystrophy News

NS Pharma has launched a support hub, called NS Support, for people in the U.S. with Duchenne muscular dystrophy (DMD) and their healthcare providers. NS Support will provide patients and clinicians ... Read more

July 14, 2020July 15, 2020

Hansa Licenses Imlifidase to Sarepta as Gene Therapy Pre-treatment for DMD, LGMD

Muscular Dystrophy News

Hansa Biopharma has granted an exclusive worldwide license to Sarepta Therapeutics to develop and promote imlifidase as a gene therapy pre-treatment for people with muscular dystrophy who would otherwise be unable ... Read more

July 10, 2020July 11, 2020

National Survey Seeks to Assess Full Social, Economic Burdens of Rare Diseases in US

Muscular Dystrophy News

The EveryLife Foundation for Rare Diseases has launched a nationwide National Burden of Rare Disease Survey to measure the full implications, economic and social, of living with rare disease in ... Read more

July 9, 2020July 9, 2020

UK Renews Puldysa Early Access Program for Duchenne Patients

Muscular Dystrophy News

Oral Puldysa (idebenone), Santhera Pharmaceuticals‘ investigational treatment for breathing problems caused by Duchenne muscular dystrophy (DMD), will be available to eligible patients in the U.K. for another year via the ... Read more

July 7, 2020July 7, 2020

FDA Asked to Approve Casimersen, Exon 45 Skipping DMD Therapy

Muscular Dystrophy News

Sarepta Therapeutics has submitted a full application to the U.S. Food and Drug Administration (FDA) seeking approval of casimersen (SRP-4045) to treat Duchenne muscular dystrophy (DMD) patients amenable to exon 45 skipping. ... Read more

July 2, 2020July 6, 2020

Coronavirus Relief Fund Created to Help DMD Patients, Families

COVID-19 News, Muscular Dystrophy News

The COVID-19 pandemic has forced many to shelter at home, caused millions of layoffs, and tightened budgets. But for families affected by Duchenne muscular dystrophy (DMD), who front extra medical ... Read more

June 30, 2020June 30, 2020

CHMP Recommends Translarna Label Update That Enables Treating DMD Patients Unable to Walk

Muscular Dystrophy News

The Committee for Medicinal Products for Human Use (CHMP), an arm of the European Medicines Agency, has recommended a label update for Translarna (ataluren) to enable its use in Duchenne muscular ... Read more