Sources and Currency Note
Specialist in Pediatric Neurology & Developmental Pediatrics
İstanbul University-Cerrahpaşa Faculty of Medicine · Nörogender Association
Last reviewed:
Intended for healthcare professionals
The text draws on established clinical knowledge in paediatric neurology together with current regulatory and scientific sources. Information in the treatment sections is current as of October 2026.
- For licensed treatments in spinal muscular atrophy and the 2026 approvals, the Muscular Dystrophy Association and the relevant regulatory announcements.
- For approved treatments in Duchenne muscular dystrophy, Parent Project Muscular Dystrophy and FDA approval timelines.
- For ataluren, the European Medicines Agency product page and the European Commission decision of 28 March 2025 not to renew the authorisation.
- For the national newborn screening programme, resources of the General Directorate of Public Health of the Turkish Ministry of Health.
- For standards of care, the Duchenne muscular dystrophy care considerations, the spinal muscular atrophy consensus care guidelines, consensus statements on congenital myopathies and congenital muscular dystrophies, and TREAT-NMD resources.
This text is a clinical reference; in the management of an individual patient it does not replace guidelines, current product information or the patient's own clinical circumstances. Treatment approvals and reimbursement conditions vary by country and are updated frequently.
Bu site yalnızca bilgilendirme amaçlıdır. İçerikler tanı, tedavi veya reçete yerine geçmez; doktorunuzun bakımının yerini almaz.