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…
Parent Project Muscular Dystrophy (PPMD) is a nonprofit that focuses on fighting for a cure, better resources, and more awareness for Duchenne muscular dystrophy (DMD). More than a year ago…
Edgewise Therapeutics has announced their plan to initiate a Phase 1 trial of EDG-5506, their small molecule therapy for Becker muscular dystrophy. It will take place at Worldwide Clinical Trials…
This year, at the 2020 CNS-ICNA Conjoint Meeting, researchers identified ataluren as a potential treatment that inhibits disease progression for patients with nonsense mutation Duchenne muscular dystrophy (nmDMD). Research suggests…
At the end of September, Sarepta Therapeutics ("Sarepta") announced positive data from a study exploring SRP-9003, a gene therapy candidate, as a potential treatment for patients with limb-girdle muscular…
The question of whether to go back to school amidst Covid-19 has been a debate across the country. Many universities have seen surges in cases, many involving students in Greek…
According to Medical Xpress, there may be a new treatment option for patients with Duchenne muscular dystrophy (DMD). Traditionally, DMD is treated with glucocorticoids. However, these often require high doses…
by Danielle Bradshaw from In The Cloud Copy Fulcrum Therapeutics, a company that focuses on clinical-stage biopharmaceuticals to improve the quality of life for patients that have rare genetic diseases,…
As sourced from BioSpace: After 44 years of the legendary Jerry Lewis hosting the Jerry Lewis MDA Telethon to bring awareness to this disorder, his tradition will live on in…
CureDuchenne Webinar: Update on the Italfarmaco Givinostat Program September 24, 2020 Hear from Dr. Paolo Bettica about the latest news related to Italfarmaco's investigational therapy givinostat, which is being tested…
Sarepta Therapeutics has met with the Office of Tissues and Advanced Therapies of the FDA in order to discuss a Type C written response about a clinical trial for SRP-9001,…
According to an article from ScienceBlog, a twist on CRISPR gene editing has allowed a team of researchers to successfully treat myotonic dystrophy type 1 in mouse models. By modifying…
According to BioSpace, the first patient has been enrolled in a trial of pamrevlumab, a Duchenne muscular dystrophy treatment. The third phase of LELANTOS plans to evaluate the drug's effects…
For twenty-five years Women of Influence has focused on empowering and helping to advance women in the workforce. Maayan Ziv, a Canadian tech entrepreneur, disability advocate, and voted one…
When it comes to researching and finding treatments for rare diseases, funding plays a crucial role. Recently, the Medical Research Future Fund (MRFF) Stem Cell Therapies Mission presented Monash…
According to Medical Xpress, a new gene therapy has been studied for the treatment of Duchenne muscular dystrophy. This treatment, SRP-9001, has been shown to deliver micro-dystrophin and improve functional…
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