According to a story from Benzinga, the pharmaceutical company BioCryst Pharmaceuticals, Inc., recently announced that its investigational product BCX7353 has been granted Fast Track Designation from the FDA for the…
According to a recent article for myscience.org, there is new potential for genetic therapy to cure the often deadly neurodegenerative disorder known as Gaucher disease. The new studies were first…
These groundbreaking research results have been published on the website of the University in Erlangen and in the New England Journal of Medicine, and I would like to share them…
We're happy to introduce a new partner to Patient Worthy's mission of advocating for rare disease patients! The Aplastic Anemia and MDS International Foundation (AAMDS) supports, connects and educates patients, caregivers…
According to a story from MPR News, Rae Baylark, who is the founder and current president of the Sickle Cell Foundation of Minnesota, felt like she had nowhere to seek…
According to a report by Science Daily, researchers recently discovered a disease in monkeys which imitates human Batten disease. Establishing the closest analog to the condition in humans, the version…
According to a Science Daily report, new research by a group in Liverpool recently uncovered a drug for the treatment of alkaptonuria. Researchers published the collaborative study in the journal…
According to a story from the pharmaceutical company Takeda, the company recently released the results from its clinical trial testing an experimental subcutaneous formula of the drug vedolizumab. The drug…
A Phase 1/2 clinical trial of the experimental drug NV1205 in patients with childhood cerebral adrenoleukodystrophy (CCALD) has been initiated, announced NeuroVia Inc. The source article can be found here,…
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