USANew treatments and approvals
Intrathecal onasemnogene abeparvovec approved in the USA for SMA patients aged 2 and older
A decision by a medicines regulator (FDA, European Commission, etc.). The regulator has reviewed the clinical studies behind the drug. For accelerated or conditional approvals, the lasting benefit is still being confirmed.
The FDA approved a new form of onasemnogene abeparvovec, given once into the spinal canal (intrathecally), for children, teenagers and adults aged 2 years and older with SMA and a confirmed mutation in the SMN1 gene. The dose does not change with age or weight. The approval is based on the STEER and STRENGTH studies; the most common side effects are upper respiratory tract infection and fever.
What does it mean for patients and families?
The gene therapy option has also opened up in the USA for older children and adults. Availability in Türkiye depends on TİTCK (Turkish Medicines and Medical Devices Agency) and SGK (Social Security Institution) processes.
Source
The source page was opened and read on October 7, 2026. Summary prepared by the Doktorclub editorial team and translated from Turkish; it has not yet been reviewed by a physician (a scientific board is being set up).
This page is translated from the Turkish original.
Related disease: Spinal muscular atrophy (SMA)
This page reports a development; it is not a treatment recommendation. Your doctor decides whether a treatment is right for you.