Study supports favorable safety and tolerability profile and clinically meaningful functional and structural benefits in ...

First patient dosed in REVEAL pivotal trial evaluating TSHA-102 (N=15, aged 6 to <22 years) in Q4 2025, with enrollment ...

KJ Muldoon is walking at home ahead of Christmas after being hospitalized last holiday due to his rare genetic disorder, CPS1 deficiency Charlotte Phillipp is a Weekend Writer-Reporter at PEOPLE. She ...

KJ Muldoon received a groundbreaking new treatment earlier this year. The baby saved from a rare disease by a first-ever personalized gene fix has reached a big milestone, taking his first steps ahead ...

A genetic disorder that leads to bleeding episodes and life-threatening infections has a new FDA approved treatment, a regulatory decision that marks two notable firsts — the first gene therapy for ...

Research links variations in the gene GRIN2A to a higher risk of developing schizophrenia and other forms of mental illness. The GRIN2A gene regulates communication between neurons by producing the ...

In a breakthrough study, scientists have discovered that a variant in one gene, GRIN2A, can directly cause mental illness – something previously believed to be the result of several mutations working ...

Revolutions in gene therapy are rapidly changing the landscape of modern medicine. Revolutions in gene therapy are rapidly changing the landscape of modern medicine, forcing society and science alike ...

Regeneron is adding its name to Tessera Therapeutics’ unique in vivo gene-writing program, inking a deal that lets it in on a prospect for alpha-1 antitrypsin deficiency (AATD) through a $150 million ...

Instead of requiring personalized gene edits for each patient, the new approach could create a standardized method to use for many diseases. By Pam Belluck and Carl Zimmer Gene-editing therapies offer ...

HANOI/SINGAPORE, Nov 19 (Reuters) - Vietnam-founded biotechnology firm Gene Solutions plans to raise $100 million through a pre-IPO round and a potential listing as soon as 2026, capitalising on ...

1 First Affiliated Hospital of Fujian Medical University, Fuzhou, China 2 Department of Neurosurgery, Binhai Branch of National Regional Medical Center, The First Affiliated Hospital, Fujian Medical ...