A new international study has shown that treating babies with spinal muscular atrophy before symptoms appear can dramatically improve outcomes.Emily ...

The FDA re-approved Wellcovorin, a drug from GSK, that the company stopped selling more than 25 years ago, as part of a push from the Trump administration to identify the causes of and potential ...

For columnist Jasmine Ramos, who's living with SMA, turning 30 prompted both excitement and uncertainty about the years ahead ...

Ten-year-old Sebastian Veiman is racing against time to receive €2.5 million gene therapy that could halt — or even reverse — ...

The FDA letter requested that a technical information update be included in the Chemistry Manufacturing and Controls (CMC) ...

Roche presented new data at ECTRIMS 2025 confirming that OCREVUS provides sustained protection against disability progression and maintains a con ...

Scientists from Tel Aviv University and Boston Children's Hospital use a optimized virus to deliver a corrected gene into the ...

Basel, 24 September 2025 - Roche (SIX: RO, ROG; OTCQX: RHHBY) presents new data for OCREVUS® (ocrelizumab) and the investigational Bruton’s tyrosine kinase (BTK) inhibitor fenebrutinib at the 41st ...

Meanwhile, U.S. drug pricing and reimbursement was an issue for 39%, while 32% of respondents to the survey, carried out by the investment bank Lazard, cited FDA regulatory uncertainty. Compared to ...

Biogen Inc. (Nasdaq: BIIB) today announced that the U.S. Food and Drug Administration (FDA) issued a Complete Response Letter (CRL) for the ...

The health regulator has requested an update to technical information on the drug, Spinraza, to be included in the Chemistry ...

Ionis Pharmaceuticals IONS announced positive topline results from a pivotal phase III study evaluating its investigational ...