ZURICH/NEW YORK, (Reuters) – Swiss drugmaker Novartis on Friday won U.S. approval for its gene therapy Zolgensma for spinal muscular atrophy (SMA), the leading genetic cause of death in infants, and priced the one-time treatment at a record $2.125 million. The... More »
NEW YORK (Reuters) – When Malachi Anderson was diagnosed with a rare and often deadly disease called spinal muscular atrophy (SMA) as an infant nearly four years ago, his parents Tina and Torence had a decision to make. In years past, many of the babies born w... More »
(Reuters) – Novartis AG, which this week announced positive interim trial results for its experimental gene therapy for spinal muscular atrophy, on Friday said investigation is underway into whether a second trial death could be related to the treatment. Novar... More »
LONDON (Reuters) – General Electric is raising its bet on biotechnology with the launch of prefabricated manufacturing units for producing virus-based gene and cell therapies, novel anti-cancer treatments and vaccines. Interest in such medicines, which use eng... More »
LONDON The world’s first life-saving gene therapy for children, developed by Italian scientists and GlaxoSmithKline, has been recommended for approval in Europe, boosting the pioneering technology to fix faulty genes. The European Medicines Agency (EMA) said o... More »
LONDON Gene therapy for deafness is moving closer to reality, with new research on Wednesday showing the technique for fixing faulty DNA can improve responses in mice with genetic hearing loss. Separately, a clinical trial backed by Novartis is under way to he... More »
