McCandless toddler with rare gene mutation cleared to come home after treatment
As of last year, Clementine was just one of 10 people who have been diagnosed with a mutation to the TLR8 gene. She received ...
AI method can spot potential disease faster, better than humans
A deep learning artificial intelligence model developed at Washington State University can identify pathology, or signs of ...
Repurposed FDA-approved drug could uncover potential treatment for rare genetic disorders
In a new study, researchers at McMaster University have identified a potential treatment for Sandhoff and Tay-Sachs ...
FiercePharma2h
PTC wins FDA approval for first brain-delivered gene therapy Kebilidi
The FDA issued its first stamp of approval for a cell or gene therapy back in 2017 to Novartis' Kymriah. | The gene therapy ...
Hosted on MSN2h
RNA Stock Hits Record High on Entering the Cardiac Disease Space
The company has decided to advance two new wholly-owned pipeline drugs, AOC 1086 and AOC 1072, targeting two rare genetic ...
FiercePharma2h
Merck joins American Lung Association in raising awareness of pulmonary arterial hypertension
As the rollout of its recently launched pulmonary arterial hypertension (PAH) drug Winrevair ramps up, Merck is continuing ...
news.ucsb.edu3h
Frog populations once decimated by disease mount a major comeback
Over 17 years, researchers and conservationists team to save, then reintroduce, mountain yellow-legged frogs to lakes in ...
BioSpace4h
PTC Wins First FDA Nod for Direct-to-Brain Gene Therapy, Targets Ultrarare Disease
The FDA has followed in the footsteps of its European counterparts and granted accelerated approval to PTC Therapeutics’ gene ...
Dallas Observer4h
Dallas Steakhouse Legend, Al Biernat, Dies at 69
On Wednesday, The Dallas Morning News reported that Dallas restauranteur Al Biernat died at 69 after a battle with ...
The Week4h
Tackling Spinal Muscular Atrophy: A long road ahead for India
Alpana Sharma, founder-director of CureSMA, a parent-led advocacy group, urges the government to include medicines for SMA in ...
News Medical6h
Study identifies a potential treatment for Sandhoff and Tay-Sachs diseases
In a groundbreaking study, researchers at McMaster University have identified a potential treatment for Sandhoff and Tay-Sachs diseases—two rare, often fatal lysosomal storage disorders that cause ...
ThePrint10h
Medicover Hospitals Successfully Performs Marathwada’s First Rare and Complex Simultaneous Liver-Kidney Transplant (SLKT)
Chhatrapati Sambhajinagar (Maharashtra) [India], November 14: Medicover Hospitals has marked a significant milestone in healthcare by successfully performing First time in Marathwada a rare and ...