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…
Funding for clinical trials can be an issue, especially for rare disease studies. Luckily, there are programs that exist to help with the financials, such as the "Clinical Studies 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…
According to a recent news item that appeared on The Denver Channel, we are now in the age of genetic research that could have an impact on the lives of…
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…
Beijing, China: According to an article in a sister publication of Sixth Tone, a woman living in China who has been treated during the past fourteen years for paroxysmal nocturnal…
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…
Happy Friday! This week we have details about an investigative therapy for Duchenne Muscular Dystrophy that has entered a phase 3 clinical trial. Also, have you ever heard of Glycogen…
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…
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…
Emflaza (deflazacort), a corticosteroid, was originally approved by the FDA in 2017 to treat Duchenne muscular dystrophy (DMD) in patients five years or older. This approval was expanded in 2019…
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…
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