Catabasis Pharmaceuticals Inc. has announced plans for a Phase 3 clinical trial called Polaris DMD. It will investigate the experimental drug edasalonexent for the treatment of patients with Duchenne muscular…
According to BioPortfolio, the company UniQure has recently announced that the first hemophilia B patient has been enrolled in their Phase III study of the treatment AMT-061, which is an…
A Phase I/II clinical trial of the investigational gene therapy SGT-001 designed to treat Duchenne muscular dystrophy has had its clinical hold lifted by the US Food and Drug Administration…
An experimental gene therapy designed to treat Duchenne muscular dystrophy has produced exciting preliminary results in a Phase 1/2a clinical trial. Sarepta Therapeutics, the company behind the therapy’s development, announced…
According to Bloomberg, there’s a new big fish in the medical technology pond. Crispr Therapeutics AG jumped in value, multiplied by six, and landed at $3 billion. How is that…
A company called Beam Therapeutics is developing a new form of genetic editing, called ‘base editing’, to treat certain genetic diseases. The full story can be read here, at MedCity…
The U.S. Food and Drug Administration (FDA) has awarded Orphan Drug Designation to the experimental drug Sarconeos, developed by the company Biophytis, for the treatment of Duchenne muscular dystrophy. The…
According to a story from wlwt.com, thirteen-year-old Diego Ramirez has been crossing the finish line at the Flying Pig Marathon for seven years, and he recently completed the finish again…
William Eames, a five-year-old boy from a village in Hertfordshire, England, was diagnosed with Duchenne muscular dystrophy when he was three. Since then, his family have worked hard to raise…
Duchenne Muscular Dystrophy (DMD) is the most common type of the 9 types of muscular dystrophy. It is a genetic disorder that causes muscle degeneration and weakness. It is a progressive…
Phillippa Farrant, whose son, Daniel, has been diagnosed with Duchenne muscular dystrophy (DMD), recently appeared in Parliament in the UK to fight for improved access to care and treatment for…
The phrase “it takes a village” gets tossed around a lot. But in Codicote, that’s what’s happening. Fundraising events and challenges are being held left and right. All of them…
Pfizer has just begun a medical trial for a potential gene therapy treatment for Duchenne muscular dystrophy, and the first patient has been dosed. Click here to read the original…
There is a growing consensus that patients need to be more included in the medical field, reports PCMA Convene. Over the last ten years increasing numbers of organisations have moved…
According to a story from genengnews.com, the pharmaceutical company Solid Biosciences is pledging to continue developing its investigation drug candidate SGT-001 for Duchenne muscular dystrophy. This is despite its clinical…
Clinical trials of a potential gene therapy treatment for Duchenne muscular dystrophy (DMD) have been put on hold after concerns emerged about the safety of the procedure, reports Genetic Engineering…
According to a story from financialbuzz.com, the biotechnology company BIOPHYTIS has submitted documentation for orphan drug designation from the European Medicines Agency (EMA). This designation will be for Sarconeos, a…
According to a story from Medical Xpress, two new treatments were able to slow down the worsening of symptoms associated with muscular dystrophy in a mouse model study. Tamoxifen and…
According to a story from sciencedaily.com, new and improved standards of treatment are allowing patients with Duchenne muscular dystrophy to have longer lifespans than in the past. By all accounts,…
According to a story from raredr.com, the FDA had to order the cessation of Phase II/III trials for the microdystrophin transfer therapy SGT-001, which was in development for the treatment…
On March 5th, Parent Project Muscular Dystrophy hosted a new kind of meeting at their annual conference. Parent Project Muscular Dystrophy (PPMD) held the meeting in Washington DC with a…
Scientists in California at Salk Institute for Biological Studies are currently developing a drug that can mimic the effects in the body usually obtained from exercise, reports the National Post.…
CRISPR gene editing technology is still mostly in its infancy, but the new tool is already causing a lot of excitement, particularly in the treatment of genetically-linked diseases. A recent…
The Imperial College of London recently began development of a body suit to improve treatment of Duchenne muscular dystrophy. The suit uses a unique artificial intelligence interface to collect and…
Last month, the pharmaceutical company Sarepta Therapeutics announced the long awaited results of a study that measured lung function in patients with Duchenne muscular dystrophy (DMD) after they took eteplirsen,…
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