Rett syndrome is a rare genetic disease affecting 1 in 10,000 newborn girls, characterized by sudden regression around 1 year ...

Discover Stoke Therapeutics' innovative RNA-based treatments like zorevunersen for Dravet syndrome, with a promising pipeline ...

Phil and Claire Drinkwater have raised more than £11,000 for charity since the loss of their 'joyful' son Jude ...

Rare diseases have become an attractive target for pharmaceutical companies both on the ASX and internationally. ... Read ...

Romi Loffler fears she will be among the hardest hit by East Renfrewshire Council's decision to means-test and charge for ...

According to the International Rett Syndrome Foundation, a diagnosis occurs approximately in one in 10,000 births worldwide. Life expectancy is also unknown, and on average, most people with the ...

Guillain-Barré Syndrome: Around 59 people were diagnosed with Guillain-Barré Syndrome (GBS) in India's Pune on Thursday (Jan 23). Around 12 out of the total infected are on ventilators due to the rare ...

Taysha Gene Therapies' TSHA-102 tackles Rett syndrome’s root cause with its miniMECP2 transgene and scAAV delivery. So far, this is TSHA’s main value driver, and it appears safe and effective ...

Zollinger-Ellison syndrome is a rare digestive disorder that results in too much gastric acid. This extra gastric acid can cause peptic ulcers in your intestine. Symptoms include abdominal pain, ...

shared a “no filter” selfie on Instagram just a few days after revealing that people who trolled her appearance online had an unexpected outcome: her Cushing syndrome diagnosis, which she ...