ReveraGen BioPharma Announces Completion of Vamorolone Study in 41 DMD Patients

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

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

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

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

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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Acceleron Pharma Announces Discontinuation of Clinical Trials Program for CMT

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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NS Pharma Launches Expanded Access Program for Viltolarsen

On March 9, NS Pharma Inc. (a wholly owned US subsidiary of Nippon Shinyaku Co. Ltd.) announced that it has launched an Expanded Access Program in the United States for viltolarsen, its investigational therapy for the treatment of Duchenne muscular dystrophy (DMD) in patients amenable to skipping exon 53. The program is open to all DMD patients . . .

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Clinical Trial Alert: Researchers at Virginia Commonwealth University Seek Participants for a LGMD2I Natural History Study

Researchers at Virginia Commonwealth University are seeking participants living with limb-girdle muscular dystrophy type 2I (LGMD2I) to participate in a natural history study. This study aims to better understand disease course so researchers will be able to determine clinically meaningful outcome measures for use in future clinical trials, including the utility of dystroglycan as a biomarker. This two-year . . .

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FDA Accepts NS Pharma’s NDA for Viltolarsen for the Treatment of DMD Amenable to Exon 53 Skipping

On Feb. 7, NS Pharma Inc. (a wholly owned US subsidiary of Nippon Shinyaku Co. Ltd.), announced that the US Food and Drug Administration (FDA) accepted its New Drug Application (NDA) seeking approval under Priority Review for viltolarsen, an investigational therapy for the treatment of Duchenne muscular dystrophy (DMD) in patients amenable to skipping exon 53. Priority . . .

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