According to a story from prnewswire.co.uk, the biopharmaceutical company PTC Therapeutics recently announced the release of initial data from the first drug registry for its product Translarna (ataluren). This registry…
According to a report by biopharmadive.com, Pfizer announced they will be dropping support for an experimental Duchenne muscular dystrophy treatment. The company intends to see if the treatment may be…
Friday September 7th is World Duchenne Muscular Dystrophy Day! Duchenne muscular dystrophy (DMD) is one of nine forms of muscular dystrophy. Patients with DMD cannot make dystrophin in their muscles causing…
According to a video and article published by Duchenne UK, Professor Steve Winder has been granted additional funds to study the effectiveness of soy as a treatment for Duchenne muscular…
This is the largest international conference dedicated to Duchenne muscular dystrophy in the world. Families affected by Duchenne muscular dystrophy will gather to share stories, network, and build community. In…
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…
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