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,…
On January 17th, Parent Project Muscular Dystrophy announced that the first patient had been treated with a new gene therapy. The therapy developed specifically for Duchenne muscular dystrophy is known as…
19-year-old Jonathan Inkin created a "20-strong" bucket list with intention to cross them all off before his birthday. Thanks to the help of celebrity Toff (Georgia Toffolo), the winner of…
Even after the third denial from the U.S. FDA, Food and Drug Administration, PTC Therapeutics Incorporation will not stop fighting for the drug Translarna that they believe will help patients…
There is potential for a new treatment for Duchenne muscular dystrophy. Duchenne muscular dystrophy (DMD) is a genetic disorder characterized by progressive muscle degeneration and weakness.To learn more about this…
Patients diagnosed with Duchenne muscular dystrophy (DMD) have always lived with a grim prognosis. Many don't make it into their 20s and face debilitating symptoms years earlier-- a dark reminder…
If you're a fan of indie documentaries and contemplating the purpose of life, you'll probably like Gabe. Long before the filming began, Gabe Weil's story started in St Louis. He…
We're in a tumultuous time for Duchenne muscular dystrophy (DMD) treatment. Recently, the DMD community was outraged when the FDA rejected ataluren (Translarna), a promising new drug, for a disease…
A spokesperson for Sarepta Therapeutics announced that their latest drug for the treatment of Duchenne muscular dystrophy is doing very well and is actually helping patients. Duchenne muscular dystrophy (DMD)…
For now, Ann Marie Harte’s five-year-old son, Lewis, can still walk. He can no longer run, and he has a hard time with the stairs. The trampoline exhausts him, and…
What can one little boy and his family do to fight a rapidly debilitating and (currently) incurable disease such as Duchenne muscular dystrophy? A lot. The diagnosis of a loved…
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