Category: News

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, Living with Muscular Dystrophy

A Letter from Case Worker Jana Unislawski to the MDA Community: How to Create an Emergency Care Plan for the COVID-19 Era

Jana Unislawski

MDA Care Center providers across the country are working on the frontlines of the novel coronavirus pandemic. Every day, they’re fielding patient questions and finding new ways to practice medicine in a new time. Today, Jana Unislawski, a clinical case worker and coordinator of the Memory Disorder Clinic at University of Florida Health, shares how . . .

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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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News, Research

Orphazyme Announces FDA Fast Track Designation for Development of Arimoclomol for Treatment of ALS

Sujatha Gurunathan

On May 22, Danish company Orphazyme announced Fast Track designation from the US Food and Drug Administration (FDA) for development of the investigational drug arimoclomol for the treatment of amyotrophic lateral sclerosis (ALS). Arimoclomol previously received Fast Track status from the FDA for the treatment of Niemann-Pick disease type C (NPC) and sporadic inclusion body . . .

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News, Living with Muscular Dystrophy

A Letter From Dr. Susan Apkon to the MDA Community: How Telehealth is Changing Medicine (and How to Prepare for It)

Susan Apkon, MD

MDA Care Center physicians across the country are working on the frontlines of the novel coronavirus pandemic. Every day, they’re fielding patient questions and finding new ways to practice medicine in a new time. Today, Dr. Susan Apkon, chief of the Department of Rehabilitation and MDA Care Center Director at Children’s Hospital Colorado, shares how . . .

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News

Acceleron Pharma Announces Discontinuation of Clinical Trials Program for CMT

Jeanene Swanson

On March 9, Acceleron Pharma, Inc., announced that a phase 2 clinical trial of its therapy under development, ACE-083, for the treatment of Charcot-Marie-Tooth disease (CMT) did not show statistically significant improvements in functional tests compared to placebo. As a result, Acceleron is discontinuing development of ACE-083 and its clinical trials program for CMT. Delivered by . . .

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