Stakeholder Collaboration for Spinal Muscular Atrophy Therapy Development
Overview
Authors
Affiliations
Brandt M, Driemeyer J, Johannsen J, Denecke J, Inhestern L, Bergelt C BMC Psychol. 2024; 12(1):566.
PMID: 39420400 PMC: 11488358. DOI: 10.1186/s40359-024-02070-4.
Brandt M, Johannsen L, Inhestern L, Bergelt C Orphanet J Rare Dis. 2022; 17(1):274.
PMID: 35854387 PMC: 9295422. DOI: 10.1186/s13023-022-02407-5.
Longitudinal natural history of type I spinal muscular atrophy: a critical review.
Mercuri E, Lucibello S, Perulli M, Coratti G, de Sanctis R, Pera M Orphanet J Rare Dis. 2020; 15(1):84.
PMID: 32248834 PMC: 7132885. DOI: 10.1186/s13023-020-01356-1.
An observational study of functional abilities in infants, children, and adults with type 1 SMA.
Pane M, Palermo C, Messina S, Sansone V, Bruno C, Catteruccia M Neurology. 2018; 91(8):e696-e703.
PMID: 30045959 PMC: 6107268. DOI: 10.1212/WNL.0000000000006050.