Fabry Disease is the number number two Lysosomal Storage disease in terms of numbers affected. It is an X-linked disease, with males passing on the mutated allele to all of their…
Current treatments for cervical dystonia provide inadequate relief to many. Dystonia twists people and leads to strange postures, involuntary movements and pain. It affects men, women and children. Check out more…
Takeda Pharmaceuticals is looking for patients to enroll in their new trial, described as; "A Phase 2, Randomized, Controlled, Open-Label, Clinical Study of the Efficacy and Safety of Pevonedistat Plus Azacitidine…
There is a new Phase 3 Clinical that is currently recruiting MDS patients, take a look at the info below if you're interested. For more info and resources about MDS…
If you've got Fabry Disease and you live in Canada, you've GOT to check this out. If you don't have Fabry Disease, allow me to clue you in: It's a…
About two months ago, I bucked up and started recycling. (No this is NOT clickbait.) Now let me first explain that for years as a “responsible adult,” I did my…
It’s always awesome to announce a new clinical trial, especially for something that could offer relief to people suffering from the rarest of the rare conditions. And rare doesn’t begin…
Chiasma is conducting another clinical trial for Acromegaly patients, the MPOWERED study. “It is designed to compare the safety and effectiveness of an investigational new drug product, octreotide capsules, to…
So this is kind of a bummer: Clinical trials looking into using Alexion Pharmaceutical’s drug Soliris for treatment-resistant generalized myasthenia gravis have just come up short of expectations. Soliris, which…
In the past, a diagnosis of Huntington's disease offered the patient absolutely no hope. It was understood that a downward trajectory was the only way the disease would play out,…
There's good news for people living with carnitine palmitoyltransferase (CPT) deficiency and their caregivers! Ultragenyx recently released data from an open-label study of the treatment triheptanoin. In the study, five infants…
In the rare disease world, nothing causes rippling waves of excitement quite like a new drug announcement. So prepare for a tsunami, because that’s exactly what you’re getting… sort of. Spring…
Quick question: What does multiple myeloma have to do with myasthenia gravis (MG)? On the surface, not much—one is a rare cancer of the blood, the other is an autoimmune…
Sometimes I hear people say “I don’t want to be a guinea pig”, “They are not going to experiment on me!”, or similar sentiments regarding medical trials. On the one…
Researchers at UCSF are searching for volunteers to participate in a clinical trial studying the effects of Deep Brain Stimulation on isolated/primary Dystonia. To participate you must meet the following…
What’s it going to take before an effective treatment gets approved by the FDA for acute and aggressive mastocytosis? Well, hopefully the mastocytosis community won’t have to wait too long! In…
Well, the French are at it again—thank goodness! French pharmaceutical company AB Science, announced in 2015 that it hit its endpoints in a phase lll clinical trial for human subjects who…
Most researchers like to say that their work is “groundbreaking” or “revolutionary.” And most reporters—whether print or digital—like to use those words too, because they help get people’s attention. But…
Mycosis Fungoides (MF) is a rare form of lymphoma that affects usually affects the skin causing inflammation, tumors, plaques, and redness. It only affects 3 to 4 people in a…
Recently, the Immune Deficiency Foundation (IDF) was invited to try for a grant sponsored by PI CONNECT. The grant would allow the IDF to study the compared effectiveness of antibiotic treatment for…
Think about it: clinical trials are like a huge strip tease. They're waiting to put it all out there, but if no one comes, it’s a huge waste of time…
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