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Golodirsen, Potential Exon 53 Skipping Therapy for Duchenne, Under Priority Review by FDA
NewsSarepta Therapeutics announced that the U.S Food and Drug Administration (FDA) has accepted its application for a priority review of golodirsen ... Read more -
Patients, Family and Friends to Take Part in Variety of Rare Disease Day Events Worldwide
NewsScores of events are afoot worldwide to mark Feb. 28, Rare Disease Day 2019. The activities aim to raise awareness about ... Read more -
Parent Project Muscular Dystrophy Takes Its ‘End Duchenne Tour’ on Road Again for 2019
NewsWith the goal of connecting with every U.S. family facing a Duchenne muscular dystrophy (DMD) diagnosis, Parent Project Muscular ... Read more -
Tips to Alleviate Symptoms of Constipation
columns, You, Me and MD - a Column by Leah LeilaniManaging one’s health while living with a disability requires much more than regular trips to the doctor and excessive ... Read more -
Duchenne Patients Again Being Treated in HOPE-2 Trial of Potential Cell Therapy, Capricor Announces
NewsCapricor Therapeutics announced that patient dosing has resumed in its HOPE-2 trial of CAP-1002, an investigative cell therapy for Duchenne ... Read more -
Defying the Odds of Living with Duchenne, Decade After Decade
NewsWhen it comes to Duchenne muscular dystrophy (DMD), Leonardo Feder knows he’s among the lucky ones. A resident of ... Read more -
PPMD Funds Gene Therapy Research Aimed at Improving Cardiac Function in DMD Patients
NewsParent Project Muscular Dystrophy (PPMD) has granted $105,000 to the University of Missouri School of Medicine to advance research into ... Read more -
I Can’t Do Everything, and That’s OK
columns, You, Me and MD - a Column by Leah LeilaniA couple weeks ago, a blogger with mitochondrial myopathy posted an interesting piece of advice in her Instagram story: “Instead ... Read more -
ICD-10 Codes, ‘Really Important’ to Rare Disease Patients, Soon Up for Fresh Consideration
NewsG71.01 is, literally, the code for Duchenne muscular dystrophy. Q93.51 stands for Angelman syndrome, and G40.419 covers generalized and ... Read more -
DMD Increases Susceptibility to Rare, Aggressive Muscle Cancer, Mouse Study Suggests
NewsSevere Duchenne muscular dystrophy (DMD) is linked to increased susceptibility to a rare and aggressive type of muscle cancer ... Read more -
FDA Revising ‘Draft Guidance’ on Developing Treatments for Rare Diseases
NewsThe U.S. Food and Drug Administration(FDA) is updating its 2015 draft guidelines for drug discovery in rare diseases, with ... Read more -
Popeyes Group Launches 15th Annual Campaign to Help MDA
NewsPopeyes Louisiana Kitchen’s franchisee Sailormen has launched the 15th Annual “Appetite for a Cure” campaign to enhance the lives of ... Read more
