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Press Releases

MDA Chief Medical and Scientific Officer Testifies at Landmark FDA Hearing for Duchenne Drug

Submitted by kjerome on Mon, 04/25/2016 - 17:33

MDA expresses enthusiasm for eteplirsen,
says treatment options need to shift from goal to reality

CHICAGO, April 25, 2016 — Speaking today before an FDA advisory committee, MDA’s chief medical and scientific officer expressed optimism that the drug eteplirsen could change the course of Duchenne muscular dystrophy and be the first of what MDA hopes will be many new treatments for MDA families.
Marathon Pharmaceuticals Presents Analyses at AAN of a Phase III Clinical Study of Deflazacort in Patients with Duchenne Muscular Dystrophy (DMD)

Submitted by corphan on Tue, 04/19/2016 - 12:10

Investigational Drug Deflazacort Has Fast Track Status, Orphan Drug Designation and Rare Pediatric Disease Designation for DMD from FDA
Vamorolone Will Advance to Phase 2 Testing for Treatment of DMD

Submitted by corphan on Mon, 03/28/2016 - 15:27

Background: ReveraGen BioPharma has announced that it has received a $3 million grant from the National Institute of Neurological Disorders and Stroke (NINDS) of the National Institutes of Health to conduct phase 2 clinical trials of vamorolone (previously VBP15) in boys with Duchenne muscular dystrophy (DMD).
Catabasis Pharmaceuticals Presents Positive CAT-1004 Data from Part A of the MoveDMDSM Trial at the 2016 Muscular Dystrophy Association Clinical Conference

Submitted by corphan on Mon, 03/21/2016 - 15:42

Full Release: http://www.catabasis.com/pdfs/CATB%20Part%20A%20data%20at%20MDA%20FINAL.pdf

- Trial Demonstrated Favorable Safety, Tolerability and Pharmacokinetics in Patients –

- Results Support Initiation of Part B of Trial, Expected in First Half of 2016 –
Marathon Pharmaceuticals Announces Nationwide Expanded Access Program for Patients with Duchenne Muscular Dystrophy (DMD)

Submitted by corphan on Thu, 03/10/2016 - 11:53

Patients Can Now Receive Deflazacort, an Investigational Treatment from Marathon, at No Cost Through Medical Research Centers Across U.S.

Full Press Release: http://www.prnewswire.com/news-releases/marathon-pharmaceuticals-announc...
MDA Shamrocks Help Fight Muscular Dystrophy

Submitted by kjerome on Tue, 03/01/2016 - 09:18

CHICAGO, March 1, 2016— Tens of thousands of retailers nationwide have rallied together to raise funds to help the Muscular Dystrophy Association (MDA) save and improve the lives of kids and adults with muscular dystrophy, ALS and related life-threatening diseases that severely weaken muscle strength and mobility by participating in the 34^th annual MDA Shamrocks program —the nation’s largest St. Patrick's Day fundraising program of its kind.

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Press Releases

MDA Chief Medical and Scientific Officer Testifies at Landmark FDA Hearing for Duchenne Drug

Marathon Pharmaceuticals Presents Analyses at AAN of a Phase III Clinical Study of Deflazacort in Patients with Duchenne Muscular Dystrophy (DMD)

Vamorolone Will Advance to Phase 2 Testing for Treatment of DMD

Catabasis Pharmaceuticals Presents Positive CAT-1004 Data from Part A of the MoveDMDSM Trial at the 2016 Muscular Dystrophy Association Clinical Conference

Marathon Pharmaceuticals Announces Nationwide Expanded Access Program for Patients with Duchenne Muscular Dystrophy (DMD)

Patients Can Now Receive Deflazacort, an Investigational Treatment from Marathon, at No Cost Through Medical Research Centers Across U.S.

MDA Shamrocks Help Fight Muscular Dystrophy

Find MDA
in your Community

Sign Up for MDA News & Updates

About Us

Press Releases

MDA Chief Medical and Scientific Officer Testifies at Landmark FDA Hearing for Duchenne Drug

Marathon Pharmaceuticals Presents Analyses at AAN of a Phase III Clinical Study of Deflazacort in Patients with Duchenne Muscular Dystrophy (DMD)

Vamorolone Will Advance to Phase 2 Testing for Treatment of DMD

Catabasis Pharmaceuticals Presents Positive CAT-1004 Data from Part A of the MoveDMDSM Trial at the 2016 Muscular Dystrophy Association Clinical Conference

Marathon Pharmaceuticals Announces Nationwide Expanded Access Program for Patients with Duchenne Muscular Dystrophy (DMD)

Patients Can Now Receive Deflazacort, an Investigational Treatment from Marathon, at No Cost Through Medical Research Centers Across U.S.

MDA Shamrocks Help Fight Muscular Dystrophy

Find MDA in your Community

Find MDA
in your Community