Quest Magazine

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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original story:

Tirasemtiv May Slow Respiratory Decline in ALS

SMA: ISIS-SMNRx Shows Benefit in Infants, Children

Update (Aug. 1, 2014): The phase 2 infant study of ISIS-SMNRx is now closed to new participants. However, a phase 3 study of this drug has opened. See ISIS-SMNRx to Be Tested in Phase 3 Trial in Infants With SMA.

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original story:

DMD: Prosensa Pursuing Path Forward for Drisapersen

Update (May 1, 2014): For encouraging results from an open-label, extension study of drisapersen (all participants receive the drug in an open-label study), see Prosensa's May 1, 2014, press release.

ALS: Experimental Drug Tirasemtiv Fails To Show Efficacy in Phase 2b Trial

Background:

 

Bret Michaels Fights Back Against Muscle Disease at Exclusive Ride for Life XXVII Performance to Benefit MDA

Shape of Genetic Material Matters in C9ORF72-Related ALS

MDA Thrilled FDA Will Consider Accelerated Approval for New Muscle Disease Drug Aimed at DMD

Dutch Bros. Coffee Annual “Drink One for Dane” Day to Donate Proceeds to Muscular Dystrophy Association

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