Tag: Research Advances

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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News, Research

Five Questions With CMT Researcher Stephan Züchner

MDA Staff

Stephan Züchner, MD, PhD, professor for Human Genetics and Neurology, Chair of the Human Genetics Department, and codirector of the John P. Hussman Institute for Human Genomics at the University of Miami Miller School of Medicine, recently discovered mutations in the sorbitol dehydrogenase gene (SORD) that cause a recessive, axonal form of Charcot-Marie-Tooth disease (CMT) . . .

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News, Research

Sarepta Therapeutics Announces Positive Results from the SRP-9003 Gene Therapy Trial to Treat LGMD2E

Sujatha Gurunathan

On June 8, Sarepta Therapeutics announced positive results from its first study in human patients of SRP-9003, a gene therapy designed to treat limb-girdle muscular dystrophy (LGMD) type 2E. Data from low-dose and high-dose patient cohorts indicated that a single intravenous infusion of SRP-9003 was well tolerated, with only one serious adverse event occurring in . . .

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News, Research

ReveraGen BioPharma Announces Completion of Vamorolone Study in 41 DMD Patients

Sujatha Gurunathan

On June 2, ReveraGen BioPharma announced the completion of two-and-a-half years of vamorolone treatment in 41 boys with Duchenne muscular dystrophy (DMD). The long-term data from this study indicated that daily oral administration of vamorolone, even at its highest tested dose, was safe and well tolerated with no serious adverse events. Preliminary data have shown . . .

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News, Research

NS Pharma Announces Positive Results from Phase 2 Study of Viltolarsen in DMD

Sujatha Gurunathan

On May 26, NS Pharma, a wholly owned subsidiary of the Japanese company Nippon Shinyaku Co. Ltd., announced positive results from its phase 2 clinical trial of the investigational therapy viltolarsen for treatment of Duchenne muscular dystrophy (DMD) amenable to skipping exon 53. The primary endpoints of the study, assessment of safety and tolerability, were . . .

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News, Research

Pfizer Announces Positive Results from Phase 1b Study of PF-06939926 for Treatment of DMD

Sujatha Gurunathan

On May 15, Pfizer announced positive results from a phase 1b trial assessing treatment with the company’s investigational gene therapy PF-06939926 in ambulatory boys with Duchenne muscular dystrophy (DMD). Preliminary data indicated that the intravenous administration of PF-06939926 was well-tolerated during the infusion period and provided improvements across multiple efficacy-related endpoints at 12 months post-infusion, . . .

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Kids, News, Research

FDA Approves PTC Therapeutics’ Emflaza for the Treatment of DMD in Patients Between 2 and 5 Years Old

Jeanene Swanson

On June 7, the U.S. Food and Drug Administration (FDA) approved Emflaza (deflazacort) to expand its labeling to include patients with Duchenne muscular dystrophy (DMD) who are between 2 and 5 years old. Emflaza was approved by the FDA in February 2017 for the treatment of DMD in patients 5 years and older, making it the first drug approved . . .

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News, Research, Advocacy

Newborn Screening for DMD and BMD: MDA Needs Your Help

MDA Staff

Early identification and treatment for neuromuscular disorders are essential to optimize health outcomes. Newborn screening, which identifies health issues via a blood test taken soon after birth, is essential to ensure that infants born with serious but treatable disorders have the best possible chance at receiving the care and support services they need as early . . .

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News, Research

FDA Grants Orphan Drug Designation to Myonexus Therapeutics’ MYO-102 Gene Therapy Drug for Limb-Girdle Muscular Dystrophy Type 2D

Jeanene Swanson

On Jan. 2, the U.S. Food and Drug Administration (FDA) awarded Orphan Drug Designation for MYO-102, an investigational gene therapy for limb-girdle muscular dystrophy type 2D (LGMD2D), also known as alpha-sarcoglycanopathy. Myonexus Therapeutics has licensed the technology from Nationwide Children’s Hospital, which holds the Investigational New Drug (IND) application for MYO-102. Treatment using gene therapy, . . .

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