Author: Jeanene Swanson

News, Research

FDA Grants Breakthrough Therapy Designation to Amicus’ Experimental Therapy, AT-GAA, for Late-Onset Pompe Disease

Jeanene Swanson

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 company’s combination drug therapy pairs ATB200, a synthetic human acid alpha-glucosidase enzyme meant to replace the missing enzyme in Pompe disease, with pharmacological chaperone AT2221, . . .

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News

FDA Accepts Sarepta Therapeutics’ New Drug Application (NDA) for Golodirsen to Treat DMD Amenable to Exon 53 Skipping

Jeanene Swanson

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 candidate for treating Duchenne muscular dystrophy (DMD) in patients amenable to skipping exon 53. Priority Review status requires the FDA to review the application and . . .

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News, Research

CMTA and MDA Co-Fund Proof-of-Concept Study Using AAV Vector to Deliver Gene Replacement Therapy in X-linked Charcot-Marie-Tooth Disease

Jeanene Swanson

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, M.D., professor and senior consulting neurologist at the Cyprus Institute of Neurology and Genetics, Cyprus School of Molecular Medicine, in Nicosia, Cyprus. Dr. Kleopa is . . .

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News, Research

Long-Term Results of Extension Study Validate Surgery as a Treatment for Myasthenia Gravis

Jeanene Swanson

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 years after surgery. The original clinical trial, which was extended for two years, showed patients who had the thymus removed had reduced muscle weakness and . . .

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News, Research

Catalyst Pharmaceuticals Announces Positive Results from Phase 2b Trial of Firdapse in Patients with Muscle-Specific Kinase Antibody Positive Myasthenia Gravis (MuSK-MG)

Jeanene Swanson

On Jan. 4, Italian researchers published positive results in the journal SAGE Open Medicine of a Phase 2b clinical trial (MSK-001) evaluating Firdapse (amifampridine phosphate) for treating MuSK antibody positive myasthenia gravis (MuSK-MG). Their results showed the drug to be safe and effective in treating patients with this rare disease. The study was partially funded . . .

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News, Research

FDA Awards Fast Track Status to RNS60, Revalesio’s Investigational Therapy for ALS

Jeanene Swanson

On Jan. 4, the U.S. Food and Drug Administration (FDA) granted Fast Track designation to Tacoma, Wash.-based Revalesio Corporation’s experimental drug for amyotrophic lateral sclerosis (ALS), RNS60.   Fast Track status helps to shorten the time it takes to develop and approve a drug that’s been designed to treat a serious condition and fill an unmet . . .

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Research

MDA Awards Venture Philanthropy Grant to AcuraStem to Develop Treatment for ALS

Jeanene Swanson

The Muscular Dystrophy Association has awarded an MDA Venture Philanthropy (MVP) grant totaling $300,000 over two years to AcuraStem to support preclinical development of a novel small molecule therapeutic for amyotrophic lateral sclerosis (ALS). The treatment has the potential to be transformative for a broad range of ALS patients. MVP grants are awarded to researchers . . .

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News, Research

Wave Life Sciences Announces Phase 2/3 Duchenne Muscular Dystrophy Clinical Trial of Suvodirsen (WVE-210201) Selected for FDA Complex Innovative Trial Designs Pilot Program

Jeanene Swanson

On Jan. 3, Wave Life Sciences announced that its upcoming Phase 2/3 clinical trial of suvodirsen (WVE-210201), an investigational therapy for boys with Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping, has been selected by the U.S. Food and Drug Administration (FDA) for its complex innovative trial designs (CID) pilot program.  Wave . . .

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News, Research

FDA Grants Orphan Drug Designation to Myonexus Therapeutics’ MYO-102 Gene Therapy Drug for Limb-Girdle Muscular Dystrophy Type 2D

Jeanene Swanson

On Jan. 2, the U.S. Food and Drug Administration (FDA) awarded Orphan Drug Designation for MYO-102, an investigational gene therapy for limb-girdle muscular dystrophy type 2D (LGMD2D), also known as alpha-sarcoglycanopathy. Myonexus Therapeutics has licensed the technology from Nationwide Children’s Hospital, which holds the Investigational New Drug (IND) application for MYO-102. Treatment using gene therapy, . . .

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