Results 131 to 140 of about 2,159 (173)

General Commentary: From hospital exemption to evidence: a statistical framework for leveraging real-world data. [PDF]

Front Immunol
Brigiari G, Muraca M, Gregori D.
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

Nusinersen: the antisense oligonucleotide at the forefront of spinal muscular atrophy treatment. [PDF]

RNA Biol
Singh NN, Ottesen EW, Singh RN.
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

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

J Prev Alzheimers Dis
Alexander RC.
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

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ZOLGENSMA-related fatal acute liver failure

Reactions Weekly, 2022
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Zolgensma-related acute liver failure: fatal cases

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Novartis Confirms Deaths of Two Patients Treated with Gene Therapy Zolgensma

Human Gene Therapy, 2022
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Amyotrophie spinale : l’affaire du zolgensma de novartis (2)

Revue Medicale Suisse, 2020
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