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Sarepta Acquires Rights to Novel Gene Therapy Candidate to Treat Limb-Girdle MD
NewsSarepta Therapeutics is acquiring an investigational gene therapy program focused on calpain-3 for the treatment of limb-girdle muscular ... Read more -
Let’s Prepare Ourselves for the Trials of a Lifetime
columns, Living, Learning, Thriving - a Column by Ralph YanizResearch and clinical trials Science is closer than ever before to finding treatments and cures for many rare ... Read more -
Rare Diseases Constitute a ‘Public Health Issue,’ NCATS Director Warns
NewsWith 250 rare diseases newly identified every year, scientists can barely keep up — even as the healthcare system ... Read more -
Duchenne Teen Hits the Runway at Las Vegas Fashion Show
NewsJoey Chorzewski has Duchenne muscular dystrophy, a neuromuscular disease that makes him different from most of his peers in ... Read more -
My Search for a Comfortable Bra as a Woman in a Wheelchair
columns, You, Me and MD - a Column by Leah LeilaniAs women, we reach a point growing up when we must enter the intimates section of the department store ... Read more -
Video Games Let Me Be Competitive Without the Need to Be Physical
columns, Hawk's-Eye View - a Column by Hawken MillerI’m not going to be an athlete — and that’s OK. I’ve found a competitive outlet through video games. ... Read more -
#AANAM – FSHD Patients ‘Very Enthusiastic’ About Taking Part in Clinical Trials, Research Suggests
NewsPeople with facioscapulohumeral muscular dystrophy (FSHD) are “very enthusiastic” about participating in clinical trials and studies, according to ... Read more -
PPMD Launches Pilot Duchenne Newborn Screening Program in NY
NewsParent Project Muscular Dystrophy (PPMD) has launched a pilot program to screen newborns in New York state for Duchenne ... Read more -
#AANAM — New TTN Gene Variants Potentially Linked to Limb-girdle MD, Study Says
NewsLimb-girdle muscular dystrophy (LGMD) may be caused by previously unidentified TTN gene variants that are also associated with ... Read more -
Find Your Communities and Gain a Sense of Belonging
columns, Living, Learning, Thriving - a Column by Ralph YanizOne of the greatest human needs is to feel that we belong. Most of us, even the introverted ones ... Read more -
#AANAM — Very Young Boys with DMD May Have Measurable Early Signs of Motor Skill Delay, Study Suggests
NewsVery young boys with Duchenne muscular dystrophy (DMD) have measurable differences in motor skills, which could have implications for ... Read more -
#AANAM — Phase 1b/2 Trial Of RG6206 in Boys With DMD Supports Safety, Positive Muscle Effects
NewsRG6206 (BMS-986089), a blocker of muscle growth-inhibitor myostatin, is safe to use in boys with Duchenne muscular dystrophy (DMD), ... Read more
