Treatment with chloroquine increased the levels and restored the activity of Muscleblind-like RNA-binding proteins, improving muscle function and strength in animal models of myotonic dystrophy type 1, a study has found. The study, “Increased Muscleblind levels by chloroquine treatment improve myotonic dystrophy type 1 phenotypes in in…
PTC Therapeutics has launched its sixth annual global grant program for patient advocacy organizations that seek to develop innovative projects for the Duchenne muscular dystrophy (DMD) community. Currently focused on DMD, the Strategies to Realize Innovation, Vision and Empowerment (STRIVE) program supports nonprofits that seek to create…
An abundance of events are afoot around the world to mark Rare Disease Day 2020 on Feb. 29. The activities are focused on heightening awareness about rare diseases and the hundreds of millions of individuals they are thought to affect. Patients, caregivers, and advocates worldwide will sport denim ribbons…
The Muscular Dystrophy Association (MDA) has launched its 38th annual #MDAShamrocks campaign — the largest St. Patrick’s fundraiser in the U.S. — aiming to raise money and awareness about muscular dystrophy and…
People known as “consumer advocates” have joined top scientists since 2011 in helping to determine how funds appropriated by the U.S. Congress will be spent on Duchenne muscular dystrophy (DMD) research. Recently, Tiffany Cook and Clint M. Porter got their turn at patient representation. Cook and Porter’s influence derived…
Registration for the upcoming Parent Project Muscular Dystrophy (PPMD) Advocacy Conference is now open. During the conference, which will be held March 8–10 in Washington, D.C., participants will receive extensive training and support to empower and prepare them to advocate for Duchenne muscular dystrophy…
Oral treatment with TAS-205 is safe, and may delay muscle deterioration in patients with Duchenne muscular dystrophy (DMD), an early Phase 2 clinical trial suggests. The findings were detailed in a study, “…
Seeking to improve treatment of Duchenne muscular dystrophy (DMD), CureDuchenne has teamed up with Vivli to make clinical trial data available to researchers around the world. The Vivli platform, which currently hosts information from more than 4,700 clinical…
The U.S. Food and Drug Administration (FDA) accepted NS Pharma‘s new drug application for viltolarsen, a potential treatment for Duchenne muscular dystrophy (DMD) patients who are amenable to exon 53 skipping, the company and its parent, Nippon Shinyaku, said. The companies expect a decision on approval between…
The U.S. Food and Drug Administration (FDA), a vast government bureaucracy, employs about 17,500 people and had a budget of $5.7 billion in 2019. Yet even with its enormous resources, the FDA these days relies more and more on patients to…
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