Results 101 to 110 of about 3,596 (160)

Addressing the Global Disparities in Access to Treatment in Spinal Muscular Atrophy. [PDF]

Muscle Nerve
Koutsioumpa C, Darras BT, Griggs RC, Roy B.   +3 more
europepmc   +1 more source

Clinical evaluation of ThecaFlex DRx, a novel implantable catheter-port for intrathecal nusinersen delivery in spinal muscular atrophy: Initial results from the PIERRE-IDE study. [PDF]

J Neuromuscul Dis
Mesina-Estarrón I, Abruzzo M, Gomes FC, Thaxton M, Yengo-Kahn A, Stone S, Northam W, Snyder B, Cahill P, Bauer D, Muhonen M.   +10 more
europepmc   +1 more source

Motor function and safety of nusinersen for spinal muscular atrophy: a systematic review and meta-analysis among Chinese patients. [PDF]

Front Pediatr
He G, Feng Z, Li N, Wei S, Liu Y, Tang X.   +5 more
europepmc   +1 more source

Comparative Characterization of a Proposed Generic Nusinersen: Identity of the Oligonucleotide Structure and Equivalence in SMN2 Splicing Activity. [PDF]

Pharmaceutics
Taran S, Smolov M, Degterev M, Lyagoskin I, Shukurov R.   +4 more
europepmc   +1 more source

Therapeutic oligonucleotides as genetic medicines: The current state and future perspective. [PDF]

Cell Rep Med
Godinho BMDC, Ferguson CM, Aartsma-Rus A.   +2 more
europepmc   +1 more source

Treatment of risdiplam after nusinersen continuously improves upper limb motor function in spinal muscular atrophy patients: a multicenter experience. [PDF]

Front Pediatr
Cheng X, Ma Y, Yu LQ, Fan YB, Zhu LH, Lu HB, Niu Q.   +6 more
europepmc   +1 more source

Glymphatic Dysfunction in Children With Type 2 and 3 Spinal Muscular Atrophy. [PDF]

CNS Neurosci Ther
Lan S, Xiang P, Yan C, Nie H, Wang H, Xu B, Yang Z, Tang W, Li Y, Liang Y, Chen Y.   +10 more
europepmc   +1 more source

MyomiR Networks in Spinal Muscular Atrophy: Associations With Clinical Severity and Treatment Response. [PDF]

Mol Neurobiol
Barbo M, Koritnik B, Leonardis L, Dolžan V, Ravnik-Glavač M.   +4 more
europepmc   +1 more source

Adherence, Persistence, and Safety of Risdiplam in Spinal Muscular Atrophy: A Population-Based Cohort Study. [PDF]

Neurol Ther
Chovi-Trull M, Ñungo-Garzón NC, Aragon-Gawinska KA, Pitarch-Castellano I, Albert-Marí A, García-Pellicer J, Poveda-Andrés JL, Edo-Solsona MD, Vázquez-Costa JF.   +8 more
europepmc   +1 more source