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Blog Post | News, Science + Research

MDA Awards More Than $1 Million in Venture Philanthropy Funding to AavantiBio to Develop Gene-Targeting Therapy for Friedreich’s Ataxia

AavantiBio, a biotechnology company developing a gene-targeting therapy for Friedreich’s ataxia (FA), was awarded MDA Venture Philanthropy (MVP) funding totaling $1,076,232 to advance AavantiBio’s phase 2 clinical trial of a…

Tags: Clinical Trials, Gene Therapy, Grants


Blog Post | News, Science + Research

FDA Approves ITF Pharma’s Tiglutik via PEG Tube for the Treatment of ALS

On Dec. 13, the US Food and Drug Administration (FDA) approved expanded labeling for ITF Pharma’s Tiglutik to include administration via percutaneous endoscopic gastrostomy (PEG) tube for the treatment of…

Tags: Drug Approval, Research Advances


Blog Post | News, Science + Research

Mallinckrodt Pharmaceuticals Releases Statement to DMD Community Announcing Discontinuation of Clinical Trials Program for DMD

Yesterday, Mallinckrodt Pharmaceuticals released a statement to the Duchenne muscular dystrophy (DMD) community regarding its decision to discontinue its DMD clinical development program. The full statement follows. After careful consideration and despite…

Tags: Clinical Trials, Research


Blog Post | News, Science + Research

Amylyx Pharmaceuticals Announces Positive Results from Phase 2 Study of AMX0035 for Treatment of ALS

On Dec. 17, Amylyx Pharmaceuticals and the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital announced positive results from the phase 2 (CENTAUR) trial assessing treatment…

Tags: Clinical Trials, Drug Development, Research, Research Advances


Blog Post | News, Science + Research

Solid Biosciences Releases Letter to DMD Community Announcing Update on IGNITE DMD Trial, Resolution of Serious Adverse Event

Today, Solid Biosciences released a letter to the Duchenne muscular dystrophy (DMD) community regarding updates on a serious adverse event (SAE) experienced by one child dosed last November and new biomarker data…

Tags: Clinical Trials, Drug Development, Research, Research Advances


Blog Post | News, Science + Research

Santhera Pharmaceuticals Announces Publication of Positive Long-Term Results from Retrospective Study of Idebenone in DMD

On Nov. 19, Santhera Pharmaceuticals announced publication of long-term data from its SYROS study showing a reduction of decline in lung function in people with Duchenne muscular dystrophy (DMD) who were…

Tags: Clinical Trials, Drug Development, Research, Research Advances


Blog Post | News, Science + Research

Wave Life Sciences Releases Letter to DMD Community Announcing Discontinuation of Clinical Trials Program for Therapies Amenable to Skipping Exons 51 and 53

Today, Wave Life Sciences released a letter to the Duchenne muscular dystrophy (DMD) community regarding its decision to discontinue its DMD clinical development program for suvodirsen, its therapy for patients amenable to…

Tags: Clinical Trials, Drug Development, Research


Blog Post | News, Science + Research

FDA Approves Sarepta Therapeutics’ Vyondys 53 for Treatment of DMD Amenable to Exon 53 Skipping

On Dec. 12, the US Food and Drug Administration (FDA) granted accelerated approval to golodirsen (Vyondys 53) for the treatment of Duchenne muscular dystrophy (DMD) in patients amenable to skipping exon…

Tags: Drug Approval, Exon Skipping, Research Advances


Blog Post | News, Science + Research

New MDA Educational Programs for Clinicians Offer a Detailed View on Selected Topics in Neuromuscular Disease

MDA’s Medical Education department was established in response to the growing need for clinician education in neuromuscular disease (NMD). We are pleased to present four webinars that cover newborn screening…

Tags: Community Education, Genetic Testing, Newborn Screening


Blog Post | News, Science + Research

FDA Approves Aquestive Therapeutics’ Exservan for the Treatment of ALS

On Nov. 25, the US Food and Drug Administration (FDA) approved riluzole oral film (Exservan) for the treatment of amyotrophic lateral sclerosis (ALS). Exservan is an oral film formulation of…

Tags: Drug Approval, Research Advances