Archives
10 Facts About FSHD — and a Reason to Smile
An older gentleman came up to me once. I had just been on TV for the Jerry Lewis MDA Labor Day Telethon talking about how facioscalpulohumeral muscular dystrophy (FSHD) affects…
Tags: Ambassadors, Community, Genetic Testing
Researcher with FSHD Awarded MDA Funding to Discover New Therapies for the Disease
Dr. Justin Cohen Like many pre-medical students, Justin Cohen discovered along the way that what he really liked was research. However, unlike others who exchange the stethoscope for a microscope,…
MDA Awards Venture Philanthropy Funding to Locana to Develop Novel Treatment for DM
Today, MDA and Locana, a leading RNA-targeting gene therapy company, announced the award of an MDA Venture Philanthropy (MVP) grant totaling $550,000 to advance Locana’s development program for myotonic dystrophy…
Tags: Drug Development, Gene Therapy, Grants, Research
FDA Approves PTC Therapeutics’ Emflaza for the Treatment of DMD in Patients Between 2 and 5 Years Old
On June 7, the U.S. Food and Drug Administration (FDA) approved Emflaza (deflazacort) to expand its labeling to include patients with Duchenne muscular dystrophy (DMD) who are between 2 and…
Tags: Drug Approval, Newborn Screening, Research
Congress is Considering a Law to Strengthen Newborn Screening: Help MDA Promote its Passage
On May 2, Reps. Lucille Roybal-Allard (CA-40), Mike Simpson (ID-02), Katherine Clark (MA-05), and Jaime Herrera-Beutler (WA-03) introduced the Newborn Screening Saves Lives Reauthorization Act of 2019, a key piece…
Clinical Trial Alert: Alexion Pharmaceuticals Seeks Participants for a Phase 3 Pediatric Myasthenia Gravis Study
Researchers at Alexion Pharmaceuticals are looking for children with generalized myasthenia gravis (gMG) to participate in an open-label Phase 3 study. The goal of the study is to evaluate the…
Clinical Trial Alert: Biogen Seeks Participants for a Phase 3 SOD1-ALS Study
Researchers at Biogen are looking for participants with amyotrophic lateral sclerosis (ALS) to participate in the VALOR Phase 3 study designed to help researchers evaluate the effects of BIIB067 on…
Do You Know ALS? Meet Dr. Cudkowicz.
Dr. Merit Cudkowicz, chief of Neurology at Massachusetts General Hospital, where the ALS Multidisciplinary Clinic in the Healey Center for ALS is associated with MDA, is dedicated to performing academic-led…
Tags: Drug Development, Grants, MDA Care Centers, Research
Do You Know ALS? Meet Tim.
In 2016, former NFL player Tim Green was diagnosed with amyotrophic lateral sclerosis (ALS) and was private about his new life at first. Over time, Tim has become an advocate…
Tags: Clinical Trials, Community, Drug Development
FDA Approves AveXis’ Zolgensma for Treatment of Spinal Muscular Atrophy in Pediatric Patients
Today, the U.S. Food and Drug Administration (FDA) approved Zolgensma (onasemnogene abeparvovac-xioi), the first gene therapy for a neuromuscular disease. Zolgensma is a one-time intravenous (into the vein) infusion for the…