Quest Magazine

DMD: Ataluren May Get Conditional Approval in Europe

The experimental Duchenne muscular dystrophy (DMD) drug ataluren (which has been given the brand name Translarna) has received encouragement from the European Medicines Agency (EMA) for conditional approval in European Union countries.

MDA Ride for Life XXVII Raises More Than $1 Million for the Fight Against Muscle Disease

Sarepta Therapeutics Announces Agreement for Acquisition of Manufacturing Facility in Massachusetts

DMD: Sarepta Will Pursue Accelerated Approval for Eteplirsen and Conduct Additional Trials

Update (May 27, 2014): Sarepta has acquired a manufacturing facility in Massachusetts to enhance its ability to produce investigational exon-skipping therapies DMD. In a May 22 press release, Sarepta CEO Chris Garabedian said, "While we scale up to address the potential U.S.

Tadalafil, Sildenafil Increase Blood Flow to DMD-Affected Muscles

A study conducted in 10 boys with Duchenne muscular dystrophy (DMD) has found that blood flow to exercising muscles is deficient and that treatment with either tadalafil (Cialis) or sildenafil (Viagra) normalizes this blood flow, at least in the short term (after one dose of either drug).

Tadalafil and sildenafil, both PDE5 inhibitors, are approved by the U.S. Food and Drug Administration (FDA) to treat erectile dysfunction.

Lowe's Raises $7.5 Million for Annual MDA Shamrocks Program

Bojangles’ Restaurants Raise More Than A Half-Million Dollars For MDA Families

A Landmark Development for the Duchenne MD Community

First Human SMA Gene Transfer Therapy Trial Opens

A phase 1 trial to test the safety and efficacy of gene transfer therapy in infants with type 1 spinal muscular atrophy (SMA) who are 9 months old or younger has opened at Nationwide Children's Hospital in Columbus, Ohio, under the direction of neurologist Jerry Mendell.

How Do Neuromuscular Disorders Affect People's Lives?

Update May 21, 2014: According to investigator Sindhu Ramchandren, 922 people had responded to this survey as of May 19, 2014. The study was closed to new participants at 5 p.m. EDT that day. At the investigator's request, the link to the online survey has been removed. Results will be announced when they become available.


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