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Press Releases

FAQ – Eteplirsen for DMD

Submitted by studiog on Fri, 02/12/2016 - 12:27

The experimental drug eteplirsen, under development by Sarepta Therapeutics to treat Duchenne muscular dystrophy (DMD), is under review by the U.S. Food and Drug Administration (FDA) for market approval. It marks an exciting and historic time for the MDA community and an important milestone for DMD treatments. Before a final decision is rendered, however, an FDA Advisory Committee will review the drug and provide a recommendation to the regulatory agency.
FDA Tells PTC Therapeutics That Ataluren (Translarna) Is Not Ready For Review At This Time

Submitted by jcook on Tue, 02/23/2016 - 14:16

Background: PTC Therapeutics today announced that it received a Refuse to File letter from the U.S. Food and Drug Administration (FDA) regarding PTC’s New Drug Application (NDA) for ataluren (brand name Translarna) to treat Duchenne muscular dystrophy (DMD).

In the letter, the FDA states that the application was not sufficiently complete to permit a substantive review. PTC is reviewing the content of the letter to determine the appropriate next steps.
Lowe’s Supports MDA Shamrocks Program and Summer Camps to Help Families and Children with Muscular Dystrophy

Submitted by studiog on Tue, 02/16/2016 - 15:49

CHICAGO, Feb. 16, 2016 — Lowe’s stores nationwide unite to participate in the Muscular Dystrophy Association’s iconic Shamrocks program now through March 31. This year marks 15 years of Lowe’s Shamrocks support that helps improve lives in communities across the country and build stronger futures for kids and adults with muscular dystrophy, ALS and related life-threatening diseases that severely weaken muscle strength and mobility.
FDA Extends PDUFA Goal Date for Eteplirsen

Submitted by kjerome on Tue, 03/22/2016 - 08:37

Background: Sarepta Therapeutics announced today that the U.S. Food and Drug Administration (FDA) has extended the PDUFA goal date for eteplirsen by a standard extension period of three months. The new date by which the FDA must make a decision about whether to approve eteplirsen for the treatment of Duchenne muscular dystrophy (DMD) is May 26, 2016.
Sailormen Inc. Launches 2016 “Appetite for a Cure” Campaign to Support Kids and Adults with Muscular Dystrophy

Submitted by studiog on Fri, 02/12/2016 - 11:56

MIAMI, Feb. 5, 2016 — Sailormen Inc., one of the largest domestic franchisees of Popeyes Louisiana Kitchen restaurants, launched its 14th annual “Appetite for a Cure” campaign on Feb. 1 that will raise critical funds and awareness to help the Muscular Dystrophy Association find urgently needed treatments and cures for muscular dystrophy, ALS and related diseases that severely limit strength and mobility.
Dosing and Enrollment in HT-100 Trial in Patients with DMD Suspended

Submitted by jcook on Mon, 05/02/2016 - 12:43

Press Release: Dosing and Enrollment in HT-100 Trial Suspended

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Press Releases

FAQ – Eteplirsen for DMD

FDA Tells PTC Therapeutics That Ataluren (Translarna) Is Not Ready For Review At This Time

Lowe’s Supports MDA Shamrocks Program and Summer Camps to Help Families and Children with Muscular Dystrophy

FDA Extends PDUFA Goal Date for Eteplirsen

Sailormen Inc. Launches 2016 “Appetite for a Cure” Campaign to Support Kids and Adults with Muscular Dystrophy

Dosing and Enrollment in HT-100 Trial in Patients with DMD Suspended

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About Us

Press Releases

FAQ – Eteplirsen for DMD

FDA Tells PTC Therapeutics That Ataluren (Translarna) Is Not Ready For Review At This Time

Lowe’s Supports MDA Shamrocks Program and Summer Camps to Help Families and Children with Muscular Dystrophy

FDA Extends PDUFA Goal Date for Eteplirsen

Sailormen Inc. Launches 2016 “Appetite for a Cure” Campaign to Support Kids and Adults with Muscular Dystrophy

Dosing and Enrollment in HT-100 Trial in Patients with DMD Suspended

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