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…
What at first glance sounded like nothing but good news, the FDA's approval of an Duchenne Muscular Dystrophy drug, has complicated undertones. It was announced last week that Emflaza (deflazacort) was approved…
If you’re like most people (especially me), you’ve never heard of nicotinamide riboside (NR) before. But that may change very soon, especially for the Duchenne muscular dystrophy (DMD) community. What…
Duchenne Muscular Dystrophy (DMD) is a degenerative disease that causes muscle weakness in the hips, thighs, shoulders and then later, in the heart and respiratory systems. This Australia family is…
Years ago, there was a television show called Tales from the Crypt. From what I remember, a creepy-looking hand would open a creaky-sounding door and the viewer would "enter" the…
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