Results 121 to 130 of about 2,159 (173)

Promises past and future - Gene therapy and the actualisation of future expectations. [PDF]

Soc Sci Med
Hilberg E, Stelmach A, Kleinhout-Vliek T, Martin P.   +3 more
europepmc   +1 more source

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

In vivo applications and toxicities of AAV-based gene therapies in rare diseases. [PDF]

Orphanet J Rare Dis
Zhao Q, Peng H, Ma Y, Yuan H, Jiang H.
europepmc   +1 more source

Genetic renaissance: a cross-regional analysis of the approval landscape of authorised gene therapeutics in paediatrics, challenges and future prospects. [PDF]

J Community Genet
Ahmad A, Simran S, Kalokhe VM, Musthafa F, Gangawane VS, Choudhary KK, Raghuvanshi RS, Srivastava S.   +7 more
europepmc   +1 more source

A Rationally Designed AAV9-DM Capsid with Minimal Liver Tropism. [PDF]

Cells
Nabakowski ZC, Jaramillo IC, Tanachaiwiwat P, Keeler GD.   +3 more
europepmc   +1 more source

When <i>pull</i>ulanase needs a little push: MyoAAV capsids enhance gene therapy for GSD IIIa. [PDF]

Mol Ther Methods Clin Dev
Venigalla S, Pacak CA.
europepmc   +1 more source

Efficacy and preliminary safety assessment of EXG001-307 AAV gene therapy for spinal muscular atrophy. [PDF]

Mol Ther Methods Clin Dev
Song C, Wang Q, Zhu P, Li J, Dai L, Hu W, Yang W, Zhang R, Wang D, Li Z, Chen S, Wang L, Ye G, Wu Z.   +13 more
europepmc   +1 more source

Meeting report: 2025 muscular dystrophy association summit on 'safety and challenges in gene therapy of neuromuscular diseases'. [PDF]

J Neuromuscul Dis
Lin BC, Lek A, Bönnemann CG, Byrne BJ.
europepmc   +1 more source

Equitable access to costly new drugs.

BMJ
Woods B, Ten Ham R, Claxton K.
europepmc   +1 more source