The Big Pharma’s window to take up exclusive rights to Taysha’s lead asset, dubbed TSHA-102, has officially closed, according ...
Today, Sammy is 17 years old and working hard to be able to walk across the stage at her high school graduation next spring. Whether kids like Sammy can achieve their dreams depends on whether ...
MONEY AND AWARENESS. CONCORD HIGH SCHOOL STUDENT IZZY BOOM IS BUSY WITH HER FRIENDS EMMA AND SAMMY. OH YEAH, I THINK THIS ONE’S MY FAVORITE DECORATING DUCKS. OH, THAT’S A CUTE ONE. TWO, TWO DUCK. BUT ...
NEW YORK and TRUMBULL, Conn., April 30, 2025 /PRNewswire/ -- Apertura Gene Therapy, a biotechnology company focused on innovative gene therapy solutions, and the Rett Syndrome Research Trust (RSRT), ...
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AI-guided drug discovery forges a novel therapeutic path for patients with Rett syndrome
Rett syndrome is a devastating rare genetic childhood disorder primarily affecting girls. Merely 1 out of 10,000 girls are born with it and much fewer boys. It is caused by mutations in the MeCP2 gene ...
COLLEGEVILLE, Pennsylvania (WPVI) -- 5-year-old Eva Rizzo has been partnered with the same horse at Sebastian Riding Associates since she was 2 years old. That became increasingly more important as ...
Researchers led by UC Davis Health scientist Sanchita Bhatnagar have developed a promising gene therapy that could treat Rett syndrome. The therapy works on reactivating healthy but silent genes ...
Accomplished nonprofit and biotech leader brings decades of rare disease experience to guide IRSF's next chapter of impact and growth. [Laura's] unique blend of scientific insight, policy experience, ...
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