Sitting in patients with spinal muscular atrophy type 1 treated with nusinersen

Newborn screening for SMA in Southern Belgium

(2019) François BOEMER et al.   NEUROMUSCULAR DISORDERS

Impact of age and motor function in a phase 1/2A study of infants with SMA Type 1 receiving single-dose gene replacement therapy

(2019) Linda P. Lowes et al.   PEDIATRIC NEUROLOGY

High-throughput genetic newborn screening for spinal muscular atrophy by rapid nucleic acid extraction from dried blood spots and 384-well qPCR

(2019) Ludwig Czibere et al.   EUROPEAN JOURNAL OF HUMAN GENETICS

Clinical Evidence Supporting Early Treatment Of Patients With Spinal Muscular Atrophy: Current Perspectives

(2019) Tamara Dangouloff et al.   Therapeutics and Clinical Risk Management

Nusinersen for SMA: expanded access programme

(2018) Michelle A Farrar et al.   JOURNAL OF NEUROLOGY NEUROSURGERY AND PSYCHIATRY

Nusinersen in type 1 SMA infants, children and young adults: Preliminary results on motor function

(2018) Marika Pane et al.   NEUROMUSCULAR DISORDERS

Nusinersen in spinal muscular atrophy type 1 patients older than 7 months

(2018) Karolina Aragon-Gawinska et al.   NEUROLOGY

Motor milestone assessment of infants with spinal muscular atrophy using the hammersmith infant neurological Exam-Part 2: Experience from a nusinersen clinical study

(2017) Kathie M. Bishop et al.   MUSCLE & NERVE

Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy

(2017) Richard S. Finkel et al.   NEW ENGLAND JOURNAL OF MEDICINE

Observational study of spinal muscular atrophy type I and implications for clinical trials

(2014) R. S. Finkel et al.   NEUROLOGY

Validation of the Childrenʼs Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND)

(2011) Allan M. Glanzman et al.   Pediatric Physical Therapy

The Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND): Test development and reliability

(2010) A.M. Glanzman et al.   NEUROMUSCULAR DISORDERS