Muscular Dystrophy Research

News, Research

MDA Funds Creation of Limb-Girdle Muscular Dystrophy Clinical Research Network to Speed Therapy Development

MDA Staff 07/27/201808/11/2018

The Muscular Dystrophy Association recently awarded an MDA clinical research network grant to Nicholas Johnson, vice chair of research and associate professor of neurology at Virginia Commonwealth University in Richmond, to establish the Limb-Girdle Muscular Dystrophy (LGMD) Clinical Research Network. The investment, totaling $700,000 over two years, supports seven centers with expertise in LGMD research . . .

News, Research

Clinical Trial Alert: Orion Pharma Seeks Participants for a Phase 3 ALS Study

MDA Staff 07/23/201808/05/2019

Researchers at Orion Pharma are looking for participants with ALS (amyotrophic lateral sclerosis) to participate in the REFALS Phase 3 study. The study is designed to help researchers evaluate the effects of oral levosimendan (ODM-109) on respiratory function in patients with ALS. Participants will be randomized to receive either levosimendan or placebo to determine whether . . .

News, Research

Clinical Trial Alert: PTC Therapeutics Seeks Participants for a Phase 3 DMD Study

MDA Staff 07/19/201807/19/2018

Researchers at PTC Therapeutics are looking for participants with Duchenne muscular dystrophy to participate in a Phase 3 study. The goal of the study is to evaluate the safety and efficacy of Translarna (ataluren). The drug is believed to promote the formation of full-length and functional dystrophin protein in boys with a nonsense (or premature . . .

News, Research

Critical Biomarker Development for CMT Receives $1 Million Boost from MDA

MDA Staff 07/09/201807/09/2018

The Muscular Dystrophy Association recently awarded an MDA human clinical trial grant for the development of a critical biomarker for Charcot-Marie-Tooth disease (CMT) to Mary M. Reilly, professor of clinical neurology and consultant neurologist, UCL Institute of Neurology and National Hospital for Neurology and Neurosurgery, London. The investment, totaling $1 million over three years, will . . .

Adults, News, Research

“Another Fatal Case of ALS”: A Message from the CDC/ATSDR

MDA Staff 06/29/201807/02/2018

Former NFL great Dwight Clark died from ALS (amyotrophic lateral sclerosis) on June 4, a little more than one year after revealing he had the disease. Clark was immortalized when he caught the winning touchdown pass in the NFC Championship Game in January 1982, sending the San Francisco 49ers to their first Super Bowl. The . . .

News, Research

Summit Therapeutics Discontinues ezutromid for DMD after Disappointing Phase 2 Trial Results

MDA Staff 06/27/201806/27/2018

This morning, Summit Therapeutics announced that it is discontinuing development of ezutromid for Duchenne muscular dystrophy after primary and secondary endpoints were missed after 48 weeks of treatment in their PhaseOut DMD trial. PhaseOut DMD was a phase 2, multi-center, open-label clinical trial of ezutromid, a utrophin-modulation therapy. Thirty-eight boys enrolled in the trial completed the 48-week regimen. The . . .

News, Research

Sarepta Reports Positive Preliminary Findings in DMD Gene Therapy Trial

MDA Staff 06/21/201806/21/2018

This week, Sarepta Therapeutics announced encouraging preliminary results from a phase 1/2a gene therapy trial designed to assess the investigational drug AAVrh74.MHCK7.micro-Dystrophin in boys with Duchenne muscular dystrophy (DMD). After 90 days in the trial, the first three participants all showed robust expression of micro-dystrophin — a shortened version of the protein that is absent . . .

News, Research

With MDA Support, Clinical Research Network is Expediting the Development of New Therapies for FSHD

Amy Madsen 06/20/201806/20/2018

The Muscular Dystrophy Association recently awarded an MDA clinical research network grant to develop and maintain a core facioscapulohumeral muscular dystrophy (FSHD) Clinical Trial Research Network (CTRN). The investment, totaling $1.2 million over three years, supports seven medical centers that specialize in FSHD research and clinical care and is targeted to spur advances in FSHD . . .

News, Research

Trial to Test Firdapse in MG Seeks Participants

Amy Madsen 05/14/201802/15/2023

Researchers are looking for people with myasthenia gravis (MG) to participate in a phase 3 clinical trial to evaluate the safety and effectiveness of the investigational drug amifampridine phosphate (brand name Firdapse). Effects of the drug will be assessed in individuals with muscle-specific kinase (MuSK) antibody positive MG and in a smaller group with acetylcholine . . .

News, Research

Trial to Test Drug Targeting Muscle Cramps in CMT Seeks Participants

Amy Madsen 05/09/201805/11/2018

Researchers are looking for people with Charcot-Marie-Tooth disease (CMT) to participate in a phase 2 clinical trial to evaluate the safety and effectiveness of the investigational drug FLX-787-ODT. FLX-787-ODT, under development by Flex Pharma, is taken in tablet form and is designed to reduce muscle cramps in adults with CMT. Trial length is approximately three . . .

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Category: Research

MDA Funds Creation of Limb-Girdle Muscular Dystrophy Clinical Research Network to Speed Therapy Development

Clinical Trial Alert: Orion Pharma Seeks Participants for a Phase 3 ALS Study

Clinical Trial Alert: PTC Therapeutics Seeks Participants for a Phase 3 DMD Study

Critical Biomarker Development for CMT Receives $1 Million Boost from MDA

“Another Fatal Case of ALS”: A Message from the CDC/ATSDR

Summit Therapeutics Discontinues ezutromid for DMD after Disappointing Phase 2 Trial Results

Sarepta Reports Positive Preliminary Findings in DMD Gene Therapy Trial

With MDA Support, Clinical Research Network is Expediting the Development of New Therapies for FSHD

Trial to Test Firdapse in MG Seeks Participants

Trial to Test Drug Targeting Muscle Cramps in CMT Seeks Participants

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