Results 131 to 140 of about 2,038 (184)

Fertility Outcomes in Risdiplam-Treated Male Patients with Spinal Muscular Atrophy: A Multicenter Case Series. [PDF]

Adv Ther
Coskery S, Erdler M, Frey MR, Lopez MA.
europepmc   +1 more source

Combinatory Treatment to Alleviate Cellular Stress and Improve Skeletal Muscle Phenotype in Spinal Muscular Atrophy. [PDF]

Circ Res
Zeng W, Kong X, Caudal A, Zhao SR, Alamana C, Melesio J, Ren L, Guzman J, Pang PD, Howell K, Chen KS, Day JW, Wu JC.   +12 more
europepmc   +1 more source

In utero genetic therapy: Treatment of early onset neurological disorders before they start. [PDF]

Mol Ther
Borges B, Brown SM, MacKenzie TC, Sumner CJ.   +3 more
europepmc   +1 more source

Post-Marketing Safety of Spinal Muscular Atrophy Therapies: Analysis of Spontaneous Adverse Drug Reactions from EudraVigilance. [PDF]

J Clin Med
Belančić A, Mas P, Miletić L, Kovačić Bytyqi B, Vitezić D.   +4 more
europepmc   +1 more source

State-of-the-Art on Model-Informed Drug Development Approaches for Pediatric Rare Diseases. [PDF]

CPT Pharmacometrics Syst Pharmacol
Krishna R, Mitra A, Zierhut ML, East L, Durairaj C.   +4 more
europepmc   +1 more source

Pre-symptomatic risdiplam treatment for spinal muscular atrophy initiated at 12 days post-birth: 14-month safety and developmental outcomes. [PDF]

Pediatr Investig
Sun Z, Wu L, Hui Y, Zhong Y, Yang J, Sun Z, Huang X, Xiong H.   +7 more
europepmc   +1 more source

A cost-utility analysis of newborn screening for spinal muscular atrophy in Canada. [PDF]

Orphanet J Rare Dis
Pace A, Roda W, Poon C, McMillan HJ, Oskoui M, MacKenzie A, Chakraborty P, Round J.   +7 more
europepmc   +1 more source

The Integrated Approach in Patients with Spinal Muscular Atrophy in the Era of Early Diagnosis, Etiopathogenic Therapies and Multidisciplinary Standards of Care and Rehabilitation Interventions Leads to New Phenotypes. [PDF]

Life (Basel)
Leanca MC, Mirea A, Nicolae G, Capitanescu A, Munteanu C, Onose G.   +5 more
europepmc   +1 more source

Transitioning From Nusinersen to Risdiplam for Spinal Muscular Atrophy in Clinical Practice: A Single-Center Experience. [PDF]

Muscle Nerve
Bekircan-Kurt CE, Subramanian S, Chagat S, Mackenzie SJ, Iammarino M, Reash N, Richardson C, Tsao CY, Noritz G, Gushue C, Kotha K, Paul G, Shell R, Alfano LN, Lowes LP, Connolly AM, Waldrop MA.   +16 more
europepmc   +1 more source

Functional and structural analysis of SITTER patients with spinal muscular atrophy. [PDF]

Sci Rep
Bieniaszewska A, Sobieska M, Gajewska E.
europepmc   +1 more source