An experimental gene therapy seems to slow the progression of Huntington’s disease by about 75 per cent, and researchers are ...

An Illinois family pushed for both boys to receive the potentially life changing medical treatment after one was approved and ...

X-linked retinoschisis gene replacement therapy, aiming to improve retinal architecture and visual function in young male ...

The UAE has become the second country worldwide to approve Itvisma, a groundbreaking gene therapy for spinal muscular atrophy ...

The first infant to undergo gene-editing therapy has accomplished another first — his first steps. KJ Muldoon is walking and ...

Explore 2025's groundbreaking scientific discoveries: the MoM-z14 galaxy, personalised CRISPR therapy, regenerative medicine, ...

Talk about a Christmas miracle!A medical breakthrough came just in time and changed one baby’s life — and gave his family a ...

Regulator approves Itvisma for adults and children aged two and above, positioning the UAE among the first countries globally to authorise the treatment for a wider age group ...

Amid news of the splashy $4.8 billion Amicus buy, BioMarin has slipped in the discontinuation of a genetic liver disease ...

Dubai, UAE: The Emirates Drug Establishment (EDE) has announced the approval of Itvisma™ (onasemnogene abeparvovec), a gene therapy that uses an adeno-associated viral vector to treat spinal muscular ...

Could one genetic mutation be sufficient to bring on complex psychopathology? For decades, dominant models of mental ...

A small group of volunteers will receive multiple injections of the experimental treatments next month, says Unlimited Bio.