Archives
FDA Grants Breakthrough Therapy Designation to Amicus’ Experimental Therapy, AT-GAA, for Late-Onset Pompe Disease
On Feb. 25, Amicus Therapeutics announced that the U.S. Food and Drug Administration (FDA) granted breakthrough therapy designation (BTD) to AT-GAA, Amicus’ investigational combination drug therapy ATB200/AT2221 for treating late-onset Pompe disease. The…
Clinical Trial Alert: Amicus Therapeutics Seeks Participants for a Phase 3 Late-Onset Pompe Disease Study
Researchers at Amicus Therapeutics are looking for adults with late-onset Pompe disease (LOPD) to participate in a Phase 3 study. The goal of the study is to evaluate the safety…
In Their Shoes: 65 Years of IAFF’s Fight for MDA
In 1952 in South Boston, Charles Crowley was desperate for help. His two sons were living with Duchenne muscular dystrophy (DMD) and needed care. The family needed resources. Charles called…
Tags: Fill the Boot, Fundraising, Summer Camp
FDA Accepts Sarepta Therapeutics’ New Drug Application (NDA) for Golodirsen to Treat DMD Amenable to Exon 53 Skipping
On Feb. 14, Sarepta Therapeutics announced that the U.S. Food and Drug Administration (FDA) accepted its New Drug Application (NDA) seeking approval under Priority Review for golodirsen (SRP-4053), its drug…
CMTA and MDA Co-Fund Proof-of-Concept Study Using AAV Vector to Deliver Gene Replacement Therapy in X-linked Charcot-Marie-Tooth Disease
On Mar. 1, the Charcot-Marie-Tooth Association (CMTA) and the Muscular Dystrophy Association (MDA) announced that they have jointly awarded a research grant totaling $276,430 over three years to Kleopas Kleopa,…
Tags: Drug Development, Gene Therapy, Grants, Research
Strengthened by Spinraza, Marley Robinson is Tackling College — and Planning for More
Feb. 28 is Rare Disease Day, when the collective rare disease community raises awareness of the conditions with which we live and advocates for access to new novel treatments like…
Tags: Ambassadors, College, Education, Young Adults
MDA Releases New DMD Fact Sheet During Duchenne Muscular Dystrophy Awareness Week
Feb. 13-18, as established by MDA’s colleagues at Parent Project Muscular Dystrophy (PPMD), is Duchenne Muscular Dystrophy Awareness Week, when we recognize both the individuals and families impacted by Duchenne…
Tags: Resources
Long-Term Results of Extension Study Validate Surgery as a Treatment for Myasthenia Gravis
Results of an extension study following an MDA-supported clinical trial showed that surgical removal of the thymus in patients with myasthenia gravis (MG) offered benefits for as long as five…
Tags: Clinical Trials, Research
Trexo Robotics Releases Trexo Home Walking Aid for Children
Joining a growing number of companies developing robotic devices for those living with mobility impairments, Ontario’s Trexo Robotics just launched the newest in its line of robotic walking aids specifically…
Tags: Innovation, Technology
Solid Biosciences Announces Preliminary Results of DMD Gene Therapy Trial
On Feb. 7, Cambridge, Mass.-based Solid Biosciences announced preliminary results from its phase 1/2 clinical trial, called IGNITE DMD, which is designed to assess the safety and efficacy of its lead…