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,…
Currently, there are no therapeutic options available for patients with myotonic dystrophy type 1 (DM1). However, that may soon change. According to Marketwatch, the FDA recently granted Fast Track…
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…
For years, researchers have evaluated Italfarmaco's givinostat for patients with Duchenne muscular dystrophy (DMD). In fact, a Phase 2/3 clinical trial showed that long-term givinostat treatment helped slow respiratory decline…
A recent study has examined what risk factors exist for patients diagnosed with myotonic dystrophy, the most common of all muscular dystrophies. This is a progressive, hereditary condition. Regarding cardiac…
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…
Genetic editing has the potential to address and treat a variety of genetic conditions. Recently, researchers explored gene editing as a potential therapeutic option for patients with facioscapulohumeral muscular dystrophy…
At the end of June 2021, pharmaceutical company Harmony Biosciences Holdings, Inc. ("Harmony") shared the initiation of a Phase 2 clinical trial via news release . During the trial, the…
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…
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…
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…
In a press release from late May 2021, AAV gene therapy company and Bayer AG subsidiary Asklepios BioPharmaceutical, Inc. ("AskBio") shared that the FDA approved its Investigational New Drug (IND)…
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…
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…
While gene therapy is a burgeoning technique to treat a number of rare genetic conditions, there are still a few complexities associated with this treatment option. How safe and effective…
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…
Colette Wheeler is one of four siblings, and although each had only a 50/50 chance of inheriting the genetic mutation that leads to facioscapulohumeral muscular dystrophy (FSHD), all of them…
Solid Biosciences has just reported that the FDA has finally lifted the hold they had placed on their Phase I/II trial for Duchenne muscular dystrophy (DMD). This trial is called…
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