Strongly

An expression of the live unlimited spirit, stories and voices of the MDA community

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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Advocacy

Clinical Trials During COVID-19: What We Need From the FDA

Paul Melmeyer

Over the course of the last several months, the US Food and Drug Administration (FDA) has undertaken many actions aimed at slowing the spread of the novel coronavirus and ensuring our first responders have the medical equipment, devices, diagnostic tools, and therapies to treat those with COVID-19. Simultaneously, the FDA has issued guidance on how . . .

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Advocacy

MDA Advocacy in These Uncertain Times

Mark Fisher

We know families across the country, especially those in the neuromuscular disease (NMD) community, are concerned about the spread of coronavirus and its associated disease, COVID-19, and the impact on their loved ones. In these uncertain times, MDA’s advocacy team will not stop serving and amplifying the voices of those in the NMD community, especially . . .

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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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News, Research

NS Pharma Launches Expanded Access Program for Viltolarsen

Jeanene Swanson

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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Muscle Walk

What’s New for MDA Muscle Walk in 2020

Lindsey Baker

Since 2011, MDA Muscle Walks have been gathering individuals living with muscular dystrophy, ALS, and related neuromuscular diseases with their families, friends, and communities across America. Together, they’ve raised awareness of everyday life with muscle disease — and they’ve raised $49 million to fund expert care and innovative research directed toward delivering cures. This year, . . .

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