If you remember reading our story on 12-year-old Markie DeVoe and his choroideremia, we have an important update for you. If you haven't read the story yet, you can find…
A new study pinpoints faulty genes as another potential cause behind hemiplegic cerebral palsy. Until now, there was an insufficient amount of information to verify that genetics was part of…
Theravance Biopharma recently proudly announced the results of a successful Phase 2b trial for their gastroparesis treatment, Velusetrag. The results showed that this treatment safely and effectively improved symptoms and normalized…
Most things have multiple functions. The tire iron you use to jack up the car when changing a flat tire? It can be used as a self-defense weapon if an…
If you attend a conference of rare disease families, you might not even notice that many of those who are helping out with all aspects of making sure that everything…
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…
Making scientific discoveries must be a lot like trying to map a coastline in the dark. There’s a lot of bumping into things and getting your feet wet in the…
Multiple Sclerosis is a disease of the brain and spinal cord. In patients with MS, their immune system attacks the protective myelin sheath that usually covers nerve fibers. These nerve…
You've got to be dedicated to train for a marathon. When Brit Joe England signed on to raise pounds for the Alkaptonuria (AKU) Society, he was optimistic about finishing the…
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