ScienceAlert24d
Fetus Receives Life-Saving Medication Before Birth in Medical First
Doctors in the US have treated a fetus with a rapidly progressive congenital disease while in the uterus for the very first ...
Hosted on MSN25d
Fetus Receives Life-Saving Medication Inside Womb in Medical First
For this single case, the FDA approved the early administration of the oral drug risdiplam, brand name Evrysdi, owned by the pharmaceutical company F. Hoffmann-La Roche AG. Over the years ...
Science Daily1mon
Promising results from first prenatal therapy for spinal muscular atrophy
Scientists at St. Jude Children's Research Hospital led the first in uterotreatment of SMA with the orally administered drug risdiplam. More than two years after the child was born, no ...
Hosted on MSN26d
Prenatal Treatment Offers Hope for Infants Born With Spinal Muscular Atrophy
So far, the child's lack of symptoms comes as a successful result of the orally administered drug risdiplam, developed by pharmaceutical company Roche, based in Basel, Switzerland. Risdiplam works ...
pharmaphorum5y
Roche edges closer to SMA market as risdiplam hits target in trial in babies
Roche is to begin discussions with regulators about new data from risdiplam, its developmental oral treatment for the rare muscle wasting disease spinal muscular atrophy (SMA) after supportive ...
Business Wire1mon
FDA Approves Genentech’s Evrysdi Tablet as First and Only Tablet for Spinal Muscular Atrophy (SMA)
About Evrysdi ® (risdiplam) Evrysdi is a survival motor neuron 2 (SMN2) pre-mRNA splicing modifier designed to treat SMA caused by mutations in chromosome 5q that lead to survival of motor neuron ...
pharmaphorum1mon
Novartis pulls rival to Roche’s risdiplam in SMA, says market limited
While Roche’s oral risdiplam has been tipped by some analysts to be a big contender in the market for spinal muscular atrophy (SMA) treatments, Novartis’ research chief Jay Bradner has a ...
Pharm Exec3d
Phase III Trial Shows Novartis’ Gene Therapy Significantly Improves Spinal Muscular Atrophy Outcomes
The open-label STRENGTH study assessed the safety, tolerability, and efficacy of OAV101 IT in 27 patients with SMA who had previously discontinued nusinersen or risdiplam. Results found that the total ...
Pharmabiz3d
Novartis announces positive data from phase III study of intrathecal onasemnogene abeparvovec in broad patient population with SMA
Novartis announces positive data from phase III study of intrathecal onasemnogene abeparvovec in broad patient population with SMA: Basel Friday, March 21, 2025, 11:00 Hrs [IST] N ...
The Hindu25d
SC stays order asking Centre to procure drugs for patient with rare disorder
The High Court on February 6 instructed that the SMA drug Risdiplam should be provided as a one-time measure to ensure continued treatment for 24-year-old Seba PA till the matter over its high ...
Deccan Herald26d
Supreme Court stays order asking Centre to procure drugs for patient with rare disorder
Seba's plea before the high court highlighted the exorbitant cost of Risdiplam, priced at Rs 6.2 lakh a bottle. PTI Last Updated : 26 February 2025, 09:37 IST ...