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…
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…
Popular wisdom tells us that the first step to fixing a problem is admitting that you have a problem. This works for individuals, like alcoholics, thanks to William Griffith Wilson,…
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,…
Duchenne muscular dystrophy is most common in males, and is characterized by muscle weakness that progresses over time. Onset is as early as three years of age and the life…
Duchenne muscular dystrophy (DMD) is a heartbreaker for the the 250,000 families worldwide whose sons are born with this debilitating, progressive disease. It is rare, and yet the ripples of its…
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…
Duchenne muscular dystrophy is a very serious and fatal disease that slowly destroys every muscle in the body. This rare disease affects one out of every 3,500 people and mostly…
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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