News

July 28, 2020July 28, 2020

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

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 ... Read more

July 23, 2020July 23, 2020

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

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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 ... Read more

July 21, 2020July 21, 2020

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

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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 ... Read more

July 16, 2020July 16, 2020

NS Pharma Opens Support Hub to Help DMD Patients Access Treatments

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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 ... Read more

July 14, 2020July 23, 2020

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

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 ... Read more

July 10, 2020July 10, 2020

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

News, syndicated

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 ... Read more

July 9, 2020July 9, 2020

UK Renews Puldysa Early Access Program for Duchenne Patients

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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 ... Read more

July 7, 2020July 20, 2020

FDA Asked to Approve Casimersen, Exon 45 Skipping DMD Therapy

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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 ... Read more

July 2, 2020July 7, 2020

Coronavirus Relief Fund Created to Help DMD Patients, Families

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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 ... Read more

June 30, 2020June 30, 2020

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

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 ... Read more

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