Swiss drugmaker Roche said 59% of infants with the severest form of spinal muscular atrophy could sit for at least five seconds after getting the company’s medicine Evrysdi for two years, according to trial data, up from 33% after a year of treatment.
Infection with the severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) during pregnancy does not appear to increase the risk of complications during delivery or negatively impact the health of newborns, according to a new JAMA study.
The U.S. Food and Drug Administration approved an oral treatment from Roche Holding AG and PTC Therapeutics Inc. for spinal muscular atrophy in adults and children two months and above.
The U.S. Food and Drug Administration approved a drug to treat infants and children with HIV, with the drug having been developed by drugmaker GlaxoSmithKline’s HIV drugs division ViiV Healthcare.
Roche company Genentech presented one-year data from FIREFISH Part 2, a pivotal trial of risdiplam in infants one to seven months old with symptomatic Type 1 spinal muscular atrophy (SMA).
Roche company Genentech announced positive top-line results from the pivotal Part 2 of the FIREFISH study looking at risdiplam in infants aged 1-7 months with Type 1 spinal muscular atrophy (SMA).
Novartis AG laid the blame for the manipulation of data behind the company’s $2.1 million gene therapy Zolgensma at the feet of the former executives Brian and Allan Kaspar.
Mallinckrodt Plc tentatively agreed to pay $15.4 million to resolve a U.S. Justice Department probe into how a drugmaker that the company now owns marketed an expensive treatment for a rare infant seizure disorder and multiple sclerosis.
San Diego-based Arcturus Therapeutics Ltd. announced that the company was reassuming 100 percent of its global rights from Germany-based CureVac AG for ARCT-810, a messenger RNA (mRNA) drug to treat OTC deficiency.
Britain’s AstraZeneca said a potential medicine to prevent respiratory syncytial virus (RSV) in babies and infants had been granted special status by U.S. and European regulators, designed to speed up the development of novel and better drugs.