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…
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…
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…
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…
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…
According to a press release put out by PTC Therapeutics, Inc., the Office of Drug Evaluation of the Food and Drug Administration on Wednesday, Oct. 25th, 2017, sent a complete…
Earlier this month, PBS released the "The Gene Doctors." The hour-long documentary follows the recent emergence of gene therapy as a novel method of treating rare diseases. The film discusses…
We've written about CRISPR technology before, and it's potential for treating sickle cell anemia. But in a latest study done at the University of California, Berkeley, sponsored by the NIH,…
Mitobridge, Inc., a pharmaceutical company that focuses on improving mitochondrial function, just announced the achievement of a vital milestone in the first-in-human clinical trial of PPAR-delta modulator, MA-0211. This study…
You’ve heard the expression, “What’s in a name?” How about, “What’s in a number?” September 7 is World Duchenne Awareness Day. It occurs on that day every year because the 7th…
Few things are scarier in life than dealing with a sick child. But for some parents, that fear is compounded into a living nightmare when they are told their child…
Happy Friday Patient Worthians! Did you know it was World PI Week this week? We have a post on the role pets have with PI. We also have a story on a…
Happy Friday Patient Worthians! PW Contributor Tom Seaman talks about what it's like to overcome the mental toll of your rare disease diagnosis. Another PW Contributor suffering from TN, dysautonomia and…
Duchenne muscular dystrophy (DMD) affects approximately 15,000 to 20,000 young boys in the US, but for the Richard and Jamie Romito family, those numbers mean nothing. All three of their sons…
I remember reading the words of Evan Davis: “It’s not a bad idea to occasionally spend a little time thinking about things you take for granted. Plain everyday things.” Some…
Greece, Italy, Egypt, Costa Rica, Dominican Republic, Mexico, Wales, Scotland, Ireland – these are all countries the Beaulieu family has explored. And the family has explored them all in the…
Duchenne muscular dystrophy (DMD) is a particularly cruel genetic disease. It most commonly affects boys, who by the age of three, begin to experience muscle weakness in the legs, hips…
A clinical trial is up for those suffering from Duchenne Muscular Dystrophy. An oral tablet called Idebenone will be compared to a placebo, to see if it is safe and…
2017 Muscular Dystrophy Association Scientific Conference was a beacon of light! Researchers from academic medical centers, pharmaceutical companies, children’s’ medical centers, biotech, engineering, and veterinary medicine came together to share…
I recently covered an author who had some solid advice for drug makers in regards to the rare disease pricing problem. The following drug maker may or may not have…
One tie, two ties, red tie, blue tie... The list goes on and on for Doug Robins, a young Australian man who’s raising awareness for Duchenne muscular dystrophy (DMD) by…
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