According to an article from rdmag.com, a new form of treatment could allow for organ tissue to regrow to a normal level in patients with conditions in which organs are…
While it's general knowledge that working out is a huge plus for a healthy life, many questioned whether it was positive or dangerous to individuals with rare diseases. Yet, BMC…
On December 11, Alexion Pharmaceuticals, Inc. released dose-ranging data from a recent clinical study involving treatment with ALXN1210, which is an investigational long-acting C5 complement inhibitor meant to treat sufferers…
A major first step for a viable treatment option! Achillion Pharmaceuticals last week reported preliminary proof-of-concept results from its ongoing Phase 2, study of a new treatment for C3 glomerulopathy…
Earlier this month, The Lancet Neurology published results from its PATH study (Polyneuropathy And Treatment with Hizentra), which showed efficacy in treating people with chronic inflammatory demyelinating polyneuropathy (CIDP). Great news and hope…
TGIF Patient Worthians! This week, we have two opportunities where you can spread some love. You can help a young boy with microcephaly and/or contribute to amyloidosis research. We also…
A biopharmaceutical company based in California called Retrophin recently announced that the first patient has been administered treatment in their phase III study in to test RE-024 (fosmetpantotenate) as a…
CymaBay, Inc. recently announced positive provisional results from its low-dose Phase II seladelpar clinical trial for primary biliary cholangitis (PBC) patients. Once you get past the confusing part of that…
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