Peeling Back the Layers of Societal Barriers for People with Disabilities
We often have the radio on at home in the mornings to keep up with news and events. I was working at my home ... Read more
Updates
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Positive Early Results Support Continuation of Phase 2 Trial of ATL1102 for DMD
Experimental therapy ATL1102 improved upper limb strength and function in six boys with Duchenne muscular dystrophy (DMD) unable to walk, according to early results ... Read more -
PPMD Grants to Promote Gene Therapy for Heart Disease and Patient Outcomes Research
Parent Project Muscular Dystrophy (PPMD) has awarded two grants, one to further development of a gene therapy to prevent heart failure linked to Duchenne ... Read more -
Catabasis Joins Jain Foundation to Advance Edasalonexent Development for Dysferlinopathy
Catabasis Pharmaceuticals and the Jain Foundation have started a preclinical research collaboration to study edasalonexent as an oral treatment candidate for dysferlinopathy, a ... Read more -
Gene Therapy SRP-9003 Showing ‘Very Encouraging’ Results at 9 Months in Limb Girdle MD, Sarepta Reports
New nine-month data from a Phase 1/2 trial show that a single administration of the gene therapy SRP-9003 (formerly, MYO-101) at low dose significantly improved functional ... Read more
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The Right Job Can Help You Find Fulfillment and Purpose
I’ve just wrapped up my internship with The Washington Post. My experience has made ... -
Request to Approve Viltolarsen, Exon 53 Skipping Treatment for Duchenne MD, Before FDA
Nippon Shinyaku filed a request for the approval of viltolarsen (NS-065/NCNP-01) to treat Duchenne muscular dystrophy ... -
First Baby Tested for Duchenne in PPMD’s Newborn Screening Pilot Program in NY
Parent Project Muscular Dystrophy (PPMD) announced the screening of a first baby for Duchenne muscular ... -
Opportunities Abound for Travel and Accessibility
My first column for Muscular Dystrophy News Today focused on the excitement of starting ... -
FDA Awards $1M to Phase 2 Study of Ifetroban in Treating Heart Disease Linked to Duchenne MD
Cumberland Pharmaceuticals announced that it has been awarded a $1 million grant by the ... -
Honoring Serena Lawrence: How Her Life Changed Mine
In the summer of 2017, I received an email from a woman named Serena ...
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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 ... Read more