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Sarepta Therapeutics Announces Positive Updated Results from Gene Therapy Trial for Duchenne Muscular Dystrophy

MDA Staff 10/11/201810/11/2018

This week, at the 23rd International Congress of the World Muscle Society in Argentina, Dr. Jerry Mendell of Nationwide Children’s Hospital in Columbus, Ohio, shared additional data relating to Sarepta Therapeutics’ AAVrh74.MHCK7.micro-Dystrophin gene therapy program for Duchenne muscular dystrophy (DMD). Dr. Mendell had previously presented results for the first three patients in the study back in . . .

News

Genentech Announces New Data for Risdiplam in SMA

MDA Staff 10/04/201810/16/2018

On Oct. 3, Genentech announced interim results from the dose-finding parts of the pivotal FIREFISH and SUNFISH studies investigating risdiplam (RG7916) in spinal muscular atrophy (SMA). Risdiplam is an investigational SMN2-splicing modifier that is given by mouth (or g-tube) and distributes widely throughout the body. Risdiplam is designed to help the SMN2 gene produce more . . .

News

FDA Announces the Approval of TIGLUTIK, an Oral Suspension Form of riluzole, for the Treatment of ALS

MDA Staff 09/10/201809/11/2018

On September 6th, the U.S. Food and Drug Administration (FDA) announced the approval of TIGLUTIK, an oral suspension form of riluzole for the treatment of amyotrophic lateral sclerosis (ALS). TIGLUTIK, marketed by ITF Pharma, the U.S. subsidiary of Italian specialty pharmaceutical company Italfarmaco, is an oral suspension formulation of riluzole, which has been available in . . .

News, Research

MDA announces 34 new research grants totaling $9.9 million

Grace Pavlath, Ph.D. 08/21/201808/23/2018

Today, MDA announced the award of 34 new grants totaling $9.9 million for its Summer 2018 grant cycle. These new grants represent a continued commitment by MDA to fund groundbreaking research that will one day lead to treatments and cures for the diseases in its program. Each grant will impact neuromuscular disease research in a . . .

News

Sarepta Therapeutics Announces Clinical Hold of Phase 1/2a DMD Micro-Dystrophin Gene Therapy Trial

MDA Staff 07/27/201807/27/2018

Late on Wednesday, Sarepta Therapeutics announced that the Food and Drug Administration (FDA) has placed a clinical hold on their Phase 1/2a Duchenne Muscular Dystrophy (DMD) Micro-Dystrophin Gene Therapy Trial due to contamination of the manufacturing lot with plasmid DNA. No safety issues were reported for participants in the trial. Sarepta has stated that they . . .

News, Advocacy

SMA Added to National List of Disorders to Screen for at Birth

Kristin Stephenson 07/05/201807/05/2018

After significant collaborative work among many stakeholders in the spinal muscular atrophy (SMA) community, we are pleased to share that the Secretary of Health and Human Services (HHS), Dr. Alex Azar, has accepted the recommendation to add SMA to the Recommended Uniform Screening Panel (RUSP) for newborns. This is a landmark decision for the SMA . . .

News, Research

13 New MDA Grants Totaling $2.6 Million Push for More Progress

Amy Madsen 04/20/201804/25/2018

MDA has announced the award of 13 new grants totaling $2.6 million as part of its continued commitment to finding treatments and cures for the diseases in its program. Each of the 13 new grants funds a project that aims to increase our understanding of the causes or underlying mechanisms of neuromuscular disease or potential . . .

News

Newborn Screening in the U.S. is One Step Closer to Including SMA

Amy Madsen 02/08/201802/08/2018

The Advisory Committee on Heritable Disorders in Newborns and Children (ACHDNC), a federal committee that oversees newborn screening, voted today to add spinal muscular atrophy (SMA) to the national list of disorders recommended for screening at birth. Following today’s vote, the Secretary of Health and Human Services (HHS) will make a final decision on whether . . .

News

A Word from MDA Chairman of the Board Dr. Rodney Howell

R. Rodney Howell 01/25/201801/25/2018

MDA’s Chairman of the Board Dr. Rodney Howell shares gratitude for the breakthroughs MDA families experienced in 2017 and looks forward to what’s to come in 2018.

News

FDA: Translarna is Not Ready for Approval at This Time

Amy Madsen 10/25/201709/08/2023

PTC Therapeutics reported today the U.S. Food and Drug Administration’s Office of Drug Evaluation is unable to approve the company’s New Drug Application for ataluren (brand name Translarna) for the treatment of Duchenne muscular dystrophy (DMD) caused by a premature stop codon, or nonsense mutation, in its current form.

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Tag: Breaking News

Sarepta Therapeutics Announces Positive Updated Results from Gene Therapy Trial for Duchenne Muscular Dystrophy

Genentech Announces New Data for Risdiplam in SMA

FDA Announces the Approval of TIGLUTIK, an Oral Suspension Form of riluzole, for the Treatment of ALS

MDA announces 34 new research grants totaling $9.9 million

Sarepta Therapeutics Announces Clinical Hold of Phase 1/2a DMD Micro-Dystrophin Gene Therapy Trial

SMA Added to National List of Disorders to Screen for at Birth

13 New MDA Grants Totaling $2.6 Million Push for More Progress

Newborn Screening in the U.S. is One Step Closer to Including SMA

A Word from MDA Chairman of the Board Dr. Rodney Howell

FDA: Translarna is Not Ready for Approval at This Time

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