EU/3/21/2456: Orphan designation for the treatment of Friedreich's ataxia. Withdrawn

Sponsor: Novartis Gene Therapies EU Limited The designation was withdrawn from the Union Register of orphan medicinal products in March 2022 upon request of the sponsor.

Neurological Recovery with Interferon-gamma Treatment in Friedreich's Ataxia

Tekin HG, Levent E.; J Coll Physicians Surg Pak. 2022 May;32(5):671-673. doi: 10.29271/jcpsp.2022.05.671. PMID: 35546709. 

The treatment was evaluated by physical examination and side effects assessment. Friedreich Ataxia Rating Scale (FARS), 9-hole peg test (9HPT), and time of 25-foot walk (T25FW) were measured. Ataxia and cerebellar findings improved within 9 months. Although clinical neurological improvement was achieved, there was no improvement in cardiomyopathy. Key Words: Interferon-gamma, Friedreich ataxia, FARS, Children, Cardiomyopathy.