EUNew treatments and approvals
EU approves single-dose gene therapy into the spinal fluid for SMA patients aged 2 years 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 European Commission approved onasemnogene abeparvovec, given into the spinal fluid, for spinal muscular atrophy. The treatment is for patients with SMA aged 2 years and older and is given once by injection in the lower back. Cortisone is given for about two months to protect the liver. The main study included 126 children who could sit but could not walk. Over one year, the motor score rose by 2.4 points with the drug and by 0.5 points with the sham procedure.
What does it mean for patients and families?
The same treatment was approved in the USA in 2025. Access in Türkiye depends on TİTCK (Turkish Medicines and Medical Devices Agency) and SGK (Social Security Institution) processes.
Source
Avrupa İlaç Ajansı (EMA) · source page
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.