Author: Sujatha Gurunathan

News

Biogen’s Early Access Program for Tofersen is Now Open for Individuals with Rapidly Progressing SOD1-ALS

Sujatha Gurunathan

Beginning in mid-July 2021, Biogen is opening part 1 of its early access program for the investigational therapy tofersen to individuals with amyotrophic lateral sclerosis (ALS) caused by mutation of the superoxide dismutase 1 gene (SOD1) who are specifically experiencing rapid disease progression. Tofersen is being investigated in a phase 3 clinical study for safety and efficacy, and the . . .

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News, Research

FDA Approves Sarepta Therapeutics’ Amondys 45 for Treatment of DMD Amenable to Skipping Exon 45

Sujatha Gurunathan

On Feb. 25, the US Food and Drug Administration (FDA) granted accelerated approval to casimersen (Amondys 45) for the treatment of Duchenne muscular dystrophy (DMD) in patients amenable to skipping exon 45. It is the fourth exon-skipping, disease-modifying drug to treat DMD, the most common childhood form of muscular dystrophy. The drug is administered by intravenous . . .

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Research

How Expedited Drug Approval Impacts the Neuromuscular Disease Treatment Landscape

Sujatha Gurunathan

The urgency of the COVID-19 pandemic has transformed the drug-development landscape, driving rapid development, US review, and, in one case, new therapeutic approval. To bring critical drugs to market, the US Food and Drug Administration (FDA) is utilizing a special emergency program announced in April 2020 to accelerate approval of promising coronavirus therapies, an initiative . . .

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Research

Once a Wild Idea, Successful First-Generation Exon-Skipping Therapies Pave the Way for Personalized Treatments

Sujatha Gurunathan

In 1996, MDA provided a small starter grant to Dr. Steve Wilton, a primary investigator at the University of Western Australia in Perth, for research into what scientists then thought was a radical process to address the effects of certain gene mutations. A quarter of a century later, this process — exon skipping — has . . .

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News, Research

Scholar Rock Announces Positive Results from Interim Analysis of TOPAZ Phase 2 Study of SRK-015 to Treat SMA

Sujatha Gurunathan

On Oct. 27, Scholar Rock announced positive results from an interim analysis of its phase 2 clinical trial evaluating SRK-015, a therapy for treating spinal muscular atrophy (SMA). After six months of treatment with SRK-015, patients with SMA types 2 and 3 demonstrated significant motor function improvements, as measured by the Hammersmith scale, with higher dose . . .

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News, Research

FDA Accepts Sarepta Therapeutics’ New Drug Application for DMD Treatment Casimersen

Sujatha Gurunathan

On Aug. 25, Sarepta Therapeutics announced that the US Food and Drug Administration (FDA) accepted the company’s New Drug Application (NDA) seeking accelerated approval for casimersen (SRP-4045), an investigational therapy for Duchenne muscular dystrophy (DMD) amenable to exon 45 skipping. The FDA has granted Sarepta’s application Priority Review Status, meaning that a decision is expected . . .

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News, Research

Biogen Announces Positive Results from Phase 1/2 Study of Tofersen to Treat a Genetic Form of ALS

Sujatha Gurunathan

On July 8, Biogen announced positive results from its phase 1/2 clinical trial of the investigational therapy tofersen (BIIB067) for treatment of amyotrophic lateral sclerosis (ALS) caused by mutation of the superoxide dismutase 1 gene (SOD1). The primary endpoints of the study — assessment of safety, tolerability, and drug dynamics in the body — demonstrated . . .

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News, Research

Sarepta Therapeutics Announces Positive Results from the SRP-9001 Gene Therapy Trial to Treat DMD

Sujatha Gurunathan

On June 15, Sarepta Therapeutics announced positive results from its phase 1/2a study of SRP-9001 gene therapy to treat Duchenne muscular dystrophy (DMD). Data from four patients indicated that a single intravenous infusion of SRP-9001 was safe and well tolerated, with no serious adverse events. Additionally, all participants demonstrated improvements across multiple efficacy-related endpoints, including . . .

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News, Research

Momenta Pharmaceuticals Announces Positive Results from Phase 2 Vivacity-MG Study of Nipocalimab for Treatment of Generalized Myasthenia Gravis

Sujatha Gurunathan

On June 15, Momenta Pharmaceuticals announced positive results from its phase 2 Vivacity-MG trial assessing treatment with the company’s investigational therapeutic nipocalimab (M281) in patients with generalized myasthenia gravis (gMG). Preliminary data indicated that treatment with nipocalimab, using four different dosing protocols over an eight-week period, resulted in improvements in patients’ Myasthenia Gravis Activities of . . .

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