Treating rare neurological diseases in children is a difficult, sometimes gut-wrenching process with no guarantees, but a ...
RWJUH and Rutgers RWJMS designated a National Rare Disease Center of Excellence from NORD, highlighting leadership in rare ...
A rare and life-threatening kidney disease in children finally has an effective therapy, thanks in large part to pioneering ...
A new AI model called popEVE can predict how likely each variant in a patient’s genome is to cause disease. The team is ...
The U.S. House of Representatives has given a unanimous thumbs up to the Give Kids A Chance Act, which would revive the FDA’s rare pediatric review voucher program following its expiration in December ...
Rare diseases, often underdiagnosed and overlooked, affect over 36 million people in the EU and around 300 million worldwide.
Acquires next generation tissue-targeted C3d-Factor H fusion protein complement inhibitor from Q32 Bio Phase 2 basket trial ...
PTC’s strong third quarter and breakout Sephience launch may help explain why one life-sciences fund is quietly upping its ...
Akebia Therapeutics has struck a deal for Q32 Bio’s deprioritized complement | Akebia Therapeutics has struck a deal for Q32 ...
Rare disease research faces complex regulations, small patient populations, and high costs. As traditional incentives wane, ...
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