Childhood-onset striatonigral degeneration is a rare genetic disorder that robs children of the ability to walk and speak by ...
Dec. 10, 2024 — In a single IV injection, a gene therapy targeting cBIN1 can reverse the effects of heart failure and restore heart function in a large animal model. The therapy increases the ...
An indigenously developed CAR T-cell gene therapy has achieved a 73% response rate for certain blood cancers in India. The ...
A baby boy with a rare illness is making “incredible” progress after he became the first ever patient to receive a new gene ...
In the time since the Novartis gene therapy Zolgensma was approved for babies with spinal muscular atrophy, other treatments ...
Gene therapy, with its offer of a possible cure for rare diseases like sickle cell, is losing early investors to ...
Although cystic fibrosis is a single gene mutation, there are more than 1,000 different ways the CFTR gene can mutate in ...
Delandistrogene moxeparvovec, a gene therapy approved for the treatment of Duchenne muscular dystrophy (DMD), was found ...
In a groundbreaking advancement for families grappling with the challenges of Dravet syndrome, a rare and life-altering form ...
An R75W mutation in the gap junction β2 (GJB2) gene causes severe fragmentation of gap junction plaques, connecting adjacent ...
With a storied history in vaccine development, India's Bharat Biotech is shaking things up and answering the call of advanced ...
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