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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