According to a recent press release from Dyne Therapeutics, the FDA has placed a clinical hold on their Investigational New Drug (IND) application for DYNE-251. Until this hold is lifted,…
According to a November 23 article from Muscular Dystrophy News, the FDA recently granted Orphan Drug designation to clinical-stage biotechnology company Regenxbio's experimental gene therapy candidate, RGX-202. The gene therapy,…
Two and a half years after announcing its launch, Dyne Therapeutics held its very first Research & Development Day. Throughout the day, there were presentations focusing on the development of…
It's a beautiful thing when a community pulls together to support one of its members. This is exactly what the Madeira community is doing for 16-year-old Diego Ramirez. He recently…
Sometimes, it can be difficult to spur drug development for rare or life-threatening diseases, considering the number of patients. To address this, and improve patient outcomes, the FDA developed the…
September 7, 2021 will be recognized as World Duchenne Awareness Day. This day will be set aside in recognition of awareness surrounding Duchenne muscular dystrophy, a rare and debilitating genetic…
On July 21, 2021, Patient Worthy attended an online webinar presentation titled "How RDCA-DAP Can Help Inform Optimal Trial Design in Progressive Rare Disease." Organized by the Critical Path Institute…
Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…
According to Muscular Dystrophy News, new research suggests that the cholesterol metabolic pathway could offer a potential therapeutic target for patients with Duchenne muscular dystrophy (DMD). Following microRNA analysis, researchers…
Clinical trial data is extremely helpful in understanding the safety, efficacy, and tolerability of various potential treatments. According to Muscular Dystrophy News, top-line data from the Phase 2b VISION-DMD…
Genome editing is a topic which has been on the forefront of medical discussion for years. After all, gene editing allows for the potential to treat - or even cure…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
Cure Rare Disease is a nonprofit organization that aims to help those who are impacted by rare diseases. Currently, the Boston-based company is planning a fundraiser to celebrate all of…
According to a story from apnews.com, the biopharmaceutical company FibroGen, Inc., has recently announced that its investigational treatment pamrevlumab has earned Rare Pediatric Disease designation from the US Food and…
Tim Revell has run the Ascension Seton Austin Marathon a whopping 15 times, with his 16th run planned for this Sunday. The father of two runs the race for his…
FibroGen has just announced that their investigative therapy pamrevlumab, for Duchenne muscular dystrophy (DMD), has received Rare Pediatric Disease designation from the FDA. The treatment already was given Fast Track…
In a recent news release, biopharmaceutical company FibroGen, Inc. ("FibroGen") shared that its therapeutic candidate, pamrevlumab, received Fast Track designation from the FDA. The anti-CTGF antibody is designed to treat…
Champions to CureDuchenne Join us for a magical evening under the stars. The event features gourmet food, cocktails, casino games, and live and silent auctions for a night of fun…
Timothy and Andrew Revell are two brothers born with Duchenne muscular dystrophy (DMD), a rare neuromuscular disorder that sees the progressive wasting and weakness of the muscles. This condition has…
Since its inception, life sciences company Solid Biosciences Inc. has been working to develop treatment options for patients with Duchenne muscular dystrophy (DMD). According to a recent press release, the…
Pamrevlumab is a first-in-class antibody that treats Duchenne muscular dystrophy (DMD) by inhibiting the effects and activity of connective tissue disorder growth factor (CTGF). It is being developed by FibroGen,…
Researchers from the Universities of Nottingham and Exeter have discovered a new, possible treatment method for Duchenne muscular dystrophy (DMD). Their research demonstrated that treating hydrogen sulfide (H2S) deficiency in…
During the virtual XVIII International Conference on Duchenne and Becker Muscular Dystrophy, specialty pharmaceutical company Italfarmaco Group ("Italfarmaco") shared updates regarding various Givinostat clinical development programs. The treatment is designed…
According to a February 25 press release, Amondys 45 (casimersen), a therapeutic option for patients with Duchenne muscular dystrophy (DMD), recently became FDA-approved. The therapy is specifically designed for patients…
According to a story from epmmagazine.com, a team of researchers from the Universities of Exeter and Nottingham have recently completely a study that could lead to the development of a…
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