Moving into a New Life with Duchenne
I recently started my internship at The Washington Post in Washington, D.C. Getting to this point was a culmination of blood, sweat, and tears ... Read more
Updates
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Popeyes’ Group Raises $681K for the MDA in 15th Annual Fundraising Drive
It’s been 15 years since Sailormen, a Popeyes Louisiana Kitchen franchise, first partnered with the Muscular Dystrophy Association (MDA) to enhance the lives of patients. ... Read more -
Eurordis Unveils Integrated-care Initiative for Rare Disease Patients
Europe’s umbrella organization for 800 rare disease associations has developed a sweeping initiative to help the continent’s 30 million rare disease patients and their ... Read more -
Biophytis Teams With AFM-Telethon to Advance Sarconeos as Duchenne MD Therapy
Biophytis SA is collaborating with AFM-Telethon to advance an oral pediatric formulation of Sarconeos (BIO101) for treating Duchenne muscular dystrophy (DMD). The pact calls for ... Read more -
Choosing an E-bike: Some Things to Consider
This column is a particularly nice one for me to write because of my love of bicycle riding. I began to bike in 1988. ... Read more
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YourDNA and Cure Rare Disease Collaborate to Raise DMD Awareness, Advance Genomics
To help make cutting-edge genetic research more accessible, and raise awareness of rare diseases ... -
The Battle Between Mitochondrial Myopathy and Makeup
My early years in this world were spent being a girlie girl. Along with ... -
Expressing Myself on YouTube with a Disability
Watching YouTube videos allows me to simultaneously learn something new, laugh out loud, and ... -
PTC Therapeutics Gets FDA Approval for Emflaza for Ages 2-5
Initially approved in the United States for Duchenne muscular dystrophy (DMD) patients age 5 ... -
DMD Patients Are Living Longer, While New Cases Hold Steady, Study Finds
The prevalence rate of Duchenne muscular dystrophy (DMD) in the United States has increased, ...
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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