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Basil T Darras

Showing results (161-170 of 222) with videos related to

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The Lancet. Child & Adolescent Health|July 1, 2026
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 trialMaria Mazurkiewicz-Bełdzińska, Laurent Servais, Giovanni Baranello, et al.
Plos One|October 19, 2020
Medical management of muscle weakness in Duchenne muscular dystrophySarah R Rivera, Sumit K Jhamb, Hoda Z Abdel-Hamid, et al.
The Lancet. Neurology|October 16, 2022
Safety and efficacy of risdiplam in patients with type 1 spinal muscular atrophy (FIREFISH part 2): secondary analyses from an open-label trialRiccardo Masson, Maria Mazurkiewicz-Bełdzińska, Kristy Rose, et al.
The Journal of Clinical Investigation|May 9, 2024
Hepatocyte-intrinsic SMN deficiency drives metabolic dysfunction and liver steatosis in spinal muscular atrophyDamien Meng-Kiat Leow, Yang Kai Ng, Loo Chien Wang, et al.
Muscle & Nerve|January 22, 2026
Liver Steatosis in Induced Hepatocytes From Carriers of Spinal Muscular AtrophyLingyu Sun, Damien Meng Kiat Leow, Loo Chien Wang, et al.
The New England Journal of Medicine|November 2, 2017
Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular AtrophyRichard S Finkel, Eugenio Mercuri, Basil T Darras, et al.
Neurogenetics|February 29, 2012
Mutations in the satellite cell gene MEGF10 cause a recessive congenital myopathy with minicoresSteven E Boyden, Lane J Mahoney, Genri Kawahara, et al.
Neurology|March 24, 2019
X-linked myotubular myopathy: A prospective international natural history studyMélanie Annoussamy, Charlotte Lilien, Teresa Gidaro, et al.
The New England Journal of Medicine|February 15, 2018
Nusinersen versus Sham Control in Later-Onset Spinal Muscular AtrophyEugenio Mercuri, Basil T Darras, Claudia A Chiriboga, et al.
Plos One|February 22, 2017
Revised Hammersmith Scale for spinal muscular atrophy: A SMA specific clinical outcome assessment toolDanielle Ramsey, Mariacristina Scoto, Anna Mayhew, et al.
Pageof 23

Showing results (161-170 of 222) with videos related to

Sort By:
Pageof 23
The Lancet. Child & Adolescent Health|July 1, 2026
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 trialMaria Mazurkiewicz-Bełdzińska, Laurent Servais, Giovanni Baranello, et al.
Plos One|October 19, 2020
Medical management of muscle weakness in Duchenne muscular dystrophySarah R Rivera, Sumit K Jhamb, Hoda Z Abdel-Hamid, et al.
The Lancet. Neurology|October 16, 2022
Safety and efficacy of risdiplam in patients with type 1 spinal muscular atrophy (FIREFISH part 2): secondary analyses from an open-label trialRiccardo Masson, Maria Mazurkiewicz-Bełdzińska, Kristy Rose, et al.
The Journal of Clinical Investigation|May 9, 2024
Hepatocyte-intrinsic SMN deficiency drives metabolic dysfunction and liver steatosis in spinal muscular atrophyDamien Meng-Kiat Leow, Yang Kai Ng, Loo Chien Wang, et al.
Muscle & Nerve|January 22, 2026
Liver Steatosis in Induced Hepatocytes From Carriers of Spinal Muscular AtrophyLingyu Sun, Damien Meng Kiat Leow, Loo Chien Wang, et al.
The New England Journal of Medicine|November 2, 2017
Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular AtrophyRichard S Finkel, Eugenio Mercuri, Basil T Darras, et al.
Neurogenetics|February 29, 2012
Mutations in the satellite cell gene MEGF10 cause a recessive congenital myopathy with minicoresSteven E Boyden, Lane J Mahoney, Genri Kawahara, et al.
Neurology|March 24, 2019
X-linked myotubular myopathy: A prospective international natural history studyMélanie Annoussamy, Charlotte Lilien, Teresa Gidaro, et al.
The New England Journal of Medicine|February 15, 2018
Nusinersen versus Sham Control in Later-Onset Spinal Muscular AtrophyEugenio Mercuri, Basil T Darras, Claudia A Chiriboga, et al.
Plos One|February 22, 2017
Revised Hammersmith Scale for spinal muscular atrophy: A SMA specific clinical outcome assessment toolDanielle Ramsey, Mariacristina Scoto, Anna Mayhew, et al.
Pageof 23