Vienna to Host RARE2019 Meeting on Rare Diseases
About 100 scientists, researchers, pharmaceutical executives, and others will converge on Austria’s capital city early next month for the 2nd International Congress on Advanced ... Read more
Updates
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MDA Releases Duchenne Fact Sheet, Survey Results Pointing to Need for Knowledge of Clinical Trials and Genetic Tests
Citing a persistent need for new treatments and patient support, the Muscular Dystrophy Association (MDA) has updated its Duchenne muscular dystrophy (DMD) fact sheet. ... Read more -
Gene Therapy SGT-001 Shows Signs of Microdystrophin Production in Muscles of Boys in IGNITE DMD Trial
Preliminary results of a Phase 1/2 study into the gene therapy candidate SGT-001 in Duchenne muscular dystrophy (DMD) patients show low levels of microdystrophin in patients’ muscles ... Read more -
Golodirsen, Potential Exon 53 Skipping Therapy for Duchenne, Under Priority Review by FDA
Sarepta Therapeutics announced that the U.S Food and Drug Administration (FDA) has accepted its application for a priority review of golodirsen (SRP-4053), a potential treatment for ... Read more -
Patients, Family and Friends to Take Part in Variety of Rare Disease Day Events Worldwide
Scores of events are afoot worldwide to mark Feb. 28, Rare Disease Day 2019. The activities aim to raise awareness about rare diseases and the millions ... Read more
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Parent Project Muscular Dystrophy Takes Its ‘End Duchenne Tour’ on Road Again for 2019
With the goal of connecting with every U.S. family facing a Duchenne muscular dystrophy ... -
Tips to Alleviate Symptoms of Constipation
Managing one’s health while living with a disability requires much more than regular trips ... -
Duchenne Patients Again Being Treated in HOPE-2 Trial of Potential Cell Therapy, Capricor Announces
Capricor Therapeutics announced that patient dosing has resumed in its HOPE-2 trial of CAP-1002, an ... -
Defying the Odds of Living with Duchenne, Decade After Decade
When it comes to Duchenne muscular dystrophy (DMD), Leonardo Feder knows he’s among the ... -
PPMD Funds Gene Therapy Research Aimed at Improving Cardiac Function in DMD Patients
Parent Project Muscular Dystrophy (PPMD) has granted $105,000 to the University of Missouri School ... -
I Can’t Do Everything, and That’s OK
A couple weeks ago, a blogger with mitochondrial myopathy posted an interesting piece of advice ...
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FDA Grants Rare Pediatric Disease Status to Myonexus’ MYO-101 for LGMD 2E
News
The U.S. Food and Drug Administration recently granted rare pediatric disease designation to Myonexus Therapeutics’ experimental gene therapy MYO-101 for the treatment of limb girdle muscular dystrophy (LGMD) type 2E, or LGMD2E. The announcement was made by Myonexus and Nationwide Children’s ... Read more