REGENXBIO Inc., a pharamaceutical company specializing in gene therapy, just shared that it launched a Investigational New Drug application to conduct Phase I/II trials on the new therapy they're developing,…
A recent study at Sanford Burnham Prebys Medical casts a ray of hope in treating multiple hereditary exostoses (MHE). MHE is a rare genetic disorder characterized by multiple noncancerous bone…
A 9-year-old boy, whose family has decided to remain anonymous, is finding relief after suffering from an incurable skin disease. According to his father, the boy was near death and…
Samuel Augustine was born 15 weeks early at Jackson Memorial Hospital with breathing problems and question marks surrounding his health. He was born with microcephaly and instantly placed on a…
Miracle workers at Queens University are working on a revolutionary method to treat patients with Lyme disease. Lyme disease is an infectious disease that springs from a tick bite's bacteria.…
Jay and Kathy Keller of Franklin, Idaho are getting creative this year with their donation efforts, reports WENY News. The Kellers are asking for people to write Christmas letters to…
Michele Holbrook is a runner-- if you spotted her jogging on the street, you wouldn't believe that her body was plagued by more than 20 tumors due to a rare…
Irish drugmaker, Shire, announced earlier this month it will bring new genetic disorder drugs to India, stepping up its worldwide presence. Shire is unique in that it's one of the…
Gideon hides it well with his infectious smile, but for over two years, he fought bravely for his life. It started when he was a mere 6-year-old toddler. Gideon had…
Pharmaceutical company, Pfizer Inc., announced recently that the U.S. FDA approved the first (and only) Janus kinase inhibitor for treating severe psoriatic arthritis (PsA). Called XELJANZ® and XELJANZ® XR, this…
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