Muna Khairi-El
Welwyn Garden City, ENG, United Kingdom
Last Updated: 09/26/2026
Save publications for later
Sign Up
Not sure about your diagnosis?
Check Your Symptoms
Found 8 publications
Efficacy and safety of risdiplam in patients with type 1 spinal muscular atrophy: a 3-year open-label extension of the two-part, phase 2 FIREFISH trial.
Journal: The Lancet. Child & adolescent health
Published: January 28, 2026
Safety and efficacy of risdiplam in patients with type 1 spinal muscular atrophy (FIREFISH part 2): secondary analyses from an open-label trial.
Journal: The Lancet. Neurology
Published: April 08, 2022
Natural history of Type 1 spinal muscular atrophy: a retrospective, global, multicenter study.
Journal: Orphanet journal of rare diseases
Published: March 08, 2022
Risdiplam-Treated Infants with Type 1 Spinal Muscular Atrophy versus Historical Controls.
Journal: The New England journal of medicine
Published: July 28, 2021
Risdiplam in Type 1 Spinal Muscular Atrophy.
Journal: The New England journal of medicine
Published: February 24, 2021
Long-term follow-up of patients with type 2 and non-ambulant type 3 spinal muscular atrophy (SMA) treated with olesoxime in the OLEOS trial.
Journal: Neuromuscular disorders : NMD
Published: July 14, 2020
High alcohol intake in deceased donors has no effect on pancreas graft survival: a registry analysis.
Journal: Transplant international : official journal of the European Society for Organ Transplantation
Published: July 25, 2016
Showing 1-8 of 8
Last Updated: 09/26/2026