Happy October! This week, we're sharing an article on a PKD treatment update. We also have news about a famous singer who shared her rare disease story recently, a piece…
On Friday, internationally renowned singer-songwriter Sia tweeted, "Hey, I'm suffering with chronic pain, a neurological disease, ehlers danlos and I just wanted to say to those of you suffering from…
According to a story from news-medical.net, a team of researchers have developed a potential new treatment for polycystic kidney disease (PKD), a rare disorder that ultimately causes organ failure. The…
According to a story from derbytelegraph.co.uk, four year old Michal Myslicki, who was born with Batten disease, will soon get access to a critical treatment that will prolong his life.…
Physician and investigator John Wood, MD, Ph.D. of Childrens Hospital of Los Angeles specializes in studying the challenges facing SCD patients. He has currently focused his attention on how oxygen delivery…
According to a story from pmlive.com, the gene therapy company Bluebird Bio's latest recently released results from the company's phase 2/3 clinical trial demonstrates further encouraging data for its Lenti-D…
According to a story from the Cystic Fibrosis Foundation's Community Blog, the internet allows for access to a trove of knowledge and information literally at your fingertips. This had naturally…
NASH Nonalcoholic Steatohepatitis (NASH) is a rare disease in which hepatic inflammation and injury to the cells occur as a result of steatosis (hepatic fat accumulation). Severe cases of NASH…
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