Results 131 to 140 of about 1,989 (180)

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

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

Equitable access to costly new drugs.

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

Unveiling trends: insights into medicines reimbursement recommendations and health technology management in Ireland (2018-2023). [PDF]

Int J Technol Assess Health Care
Aluga D, Gorry C, Barry M, Byrne P, Gillespie P, Smith SM.   +5 more
europepmc   +1 more source

Preclinical efficacy of a gene therapy for <i>CHKB</i>-mediated muscular dystrophy. [PDF]

Mol Ther Adv
Tavasoli M, Alkandari M, Dorighello G, Devitt J, Hagerty L, Damsker J, Hoffman EP, McMaster CR.   +7 more
europepmc   +1 more source

Spinal muscular atrophy in the era of newborn screening: how the classification could change. [PDF]

Front Neurol
Varone A, Esposito G, Bitetti I.
europepmc   +1 more source

Gene and genetic therapies in Alzheimer's disease and other dementias. [PDF]

J Prev Alzheimers Dis
Alexander RC.
europepmc   +1 more source

Nonclinical toxicity study duration in AAV gene therapy development: Evidence from industry survey supports adequacy of short-term assessments. [PDF]

Mol Ther Methods Clin Dev
Assaf BT, Edwards D, Berens SJ, Lauritzen B, Pierrot C, Tukov FF, Bertinetti-Lapatki C, Braun M, Schaefer K, Lynch JL.   +9 more
europepmc   +1 more source