April 15th is International Pompe Disease Day! The International Pompe Association (IPA) is an organization of Pompe disease patient groups that seeks to coordinate activities and share experiences worldwide. The…
According to a story from Rocket News, the class of drugs known for their "gene silencing" mechanism of action appear to be effective in treating porphyria, a rare disease that…
It's one thing to cope with a new rare disease diagnosis. It's another entirely to cope with that diagnosis and then four years later learn that diagnosis is incorrect and…
Biophosphonates (BPs) are common treatments for osteoporosis and metastatic bone cancer. They help to prevent bone loss. Unfortunately, these drugs can have serious side effects. One of the most severe…
Danon Disease Danon disease (DD) is an extremely rare condition that is not very well understood in the medical community. It was first described in 1981, and it is named…
A three-year research grant of $11 million has been awarded through the Gilbert Family Foundation in America. According to a recent article in News-Medical Life Sciences, the Foundation was formed…
According to a story from CRWE World, the genetic medicines company Audentes Therapeutics has recently announced its intent to develop new therapies for myotonic dystrophy and Duchenne muscular dystrophy. These…
According to a press release from Inovio Pharmaceuticals, the American biotech company's experimental recurrent respiratory papillomatosis (RRP) drug INO-3106 showed promising results at the end of a pilot clinical study…
According to a story from finanzen.at, the biotechnology company Orbsen Therapeutics recently announced that its immunotherapy product ORBCEL-C is to be tested in a multi-site clinical trial called MERLIN that…
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