Day 25 of 30 Days of MD: Cannabis and MD
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Day 25 of 30 Days of MD: Cannabis and MD
Final Tests Planned into Potential Gene Therapy for OPMD, Could Support Trial in Patients
Three new animal-model tests of a potential gene therapy for oculopharyngeal muscular dystrophy (OPMD), known as BB-301, are planned, Benitec Biopharma announced. Results are expected to support a request for a Phase 1 clinical trial in patients. OPMD results from a faulty PABPN1 gene, leading to a protein…
September 24, 2019
by Ralph Yaniz
Day 24 of 30 Days of MD: Travel Resources
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Redefining the Word ‘Independence’
Since early childhood, we have all strived to become independent. We want to be able to do things on our own. However, to achieve independence with Duchenne muscular dystrophy, we must flip the typical definition of the word on its head. When we think about…
CureDuchenne Offers One-Stop Informative Guide on Durable Medical Equipment
To help families find the ideal equipment at every stage of need for those with Duchenne muscular dystrophy (DMD), CureDuchenne launched on its website a comprehensive and interactive guide. Presenting the latest offerings together in one place, the digital Durable Medical Equipment Guide (DME) is meant to reduce frustration…
September 23, 2019
by Danielle "Dani" Liptak
Day 23 of 30 Days of MD: Alternative Therapies and MD
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September 22, 2019
by Ralph Yaniz
Day 22 of 30 Days of MD: Silly Comments/Questions
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September 21, 2019
by Danielle "Dani" Liptak
Day 21 of 30 Days of MD: Pinball and MD
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September 20, 2019
by Ralph Yaniz
Day 20 of 30 Days of MD: Recognizing Disability Rights
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Suvodirsen Receives FDA’s Fast Track Status to Treat DMD
The U.S. Food and Drug Administration (FDA) has granted fast track designation to suvodirsen as a treatment for people with Duchenne muscular dystrophy (DMD). The agency’s decision — which will expedite the review process for suvodirsen — was based on experimental and preclinical data demonstrating the treatment’s…
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