Rare inherited diseases merit disease-specific trials

Affiliations

¹ APHP, Department of Genetics, Inserm U 1127, CNRS UMR 7225, Sorbonne Universités, UPMC Univ Paris 06 UMR S 1127, Institut du Cerveau et de la Moelle Épinière, ICM, University Hospital Pitié-Salpêtrière, Paris, France. Electronic address: alexandra.durr@upmc.fr.

Comment

Alexandra Durr. Lancet Neurol. 2015 Oct.

. 2015 Oct;14(10):968-9.

doi: 10.1016/S1474-4422(15)00217-3. Epub 2015 Aug 25.

¹ APHP, Department of Genetics, Inserm U 1127, CNRS UMR 7225, Sorbonne Universités, UPMC Univ Paris 06 UMR S 1127, Institut du Cerveau et de la Moelle Épinière, ICM, University Hospital Pitié-Salpêtrière, Paris, France. Electronic address: alexandra.durr@upmc.fr.

No abstract available

Comment on

Riluzole in patients with hereditary cerebellar ataxia: a randomised, double-blind, placebo-controlled trial.
Romano S, Coarelli G, Marcotulli C, Leonardi L, Piccolo F, Spadaro M, Frontali M, Ferraldeschi M, Vulpiani MC, Ponzelli F, Salvetti M, Orzi F, Petrucci A, Vanacore N, Casali C, Ristori G. Romano S, et al. Lancet Neurol. 2015 Oct;14(10):985-91. doi: 10.1016/S1474-4422(15)00201-X. Epub 2015 Aug 25. Lancet Neurol. 2015. PMID: 26321318 Clinical Trial.