Results 101 to 110 of about 2,204 (179)

Enabling manufacture and access to advanced therapy medicinal products in low- and middle-income countries. [PDF]

Hlife
Campos de Carvalho AC, Sachetti CG, Krieger MA.   +2 more
europepmc   +1 more source

Summary of Research: Risdiplam Treatment Following Onasemnogene Abeparvovec in Individuals with Spinal Muscular Atrophy: A Multicenter Case Series. [PDF]

Adv Ther
Svoboda MD, Kuntz N, Leon-Astudillo C, Byrne BJ, Krueger J, Kwon JM, Sieburg C, Castro D.   +7 more
europepmc   +1 more source

Advanced therapy medicinal products in Saudi Arabia: Regulation and development. [PDF]

Regen Ther
AlMuhanna MH, Alharbi M, Aldahash A, Felemban R, Asiri L, Alghamdi M, Alzahrani R, Aldossary W, Alenzi N, Almohaywi M.   +9 more
europepmc   +1 more source

Zolgensma e Atrofia Muscular Espinhal: evidências, incertezas e impacto orçamentário

Primeiro Congresso da REBRATS, 2019
Kennedy Crepalde Ribeiro, Isabella Zuppo, Natália Dias Brandão, Nelio Gomes Ribeiro Junior, Pâmela Santos Azevedo, Ludmila Peres Gargano, Augusto Afonso Guerra Junior, FRANCISCO ACURCIO, Juliana Alvares-Teodoro   +8 more
openaire   +2 more sources

Vulnerability, protection and fairness: ethical and regulatory limits in small-N gene therapy trials. [PDF]

Trials
Reckelbus M, Van Steijvoort E, Huys I, Borry P.   +3 more
europepmc   +1 more source

Newborn Screening for Spinal Muscular Atrophy in the UK: Use of Modelling to Identify Priorities for Ongoing Evaluation. [PDF]

Int J Neonatal Screen
Thokala P, Bessey A, Knowles R, Marshall J, Visintin C, Lawton M, Lombardo S.   +6 more
europepmc   +1 more source

The origins of Novo Nordisk and Novartis products: piloting a framework to identify the public contributions. [PDF]

J Pharm Policy Pract
Fabian D, Sehic O, Wild C.
europepmc   +1 more source

Examination of the Peripheral Nervous System in Children With Spinal Muscular Atrophy: A High-Resolution Ultrasonographic Study. [PDF]

Brain Behav
Wurster J, West E, Meier S, Bürke NP, Jansen L, Broser PJ.   +5 more
europepmc   +1 more source

Génterápiák bemutatása farmakológiai szemmel

A génterápia a modern orvoslás egyik legintenzívebben fejlődő és legígéretesebb területe, amely lehetőséget kínál mind genetikai, mind nem genetikai betegségek oki kezelésére.
Bódi, Ádám
core  

[Spinal muscular atrophy: Clinical and genetic aspects, and therapeutic alternatives]. [PDF]

Rev Med Inst Mex Seguro Soc
González-Morales IJ, Olivera-Bernal GC, León-González C DA, Rosas-Vargas H.   +3 more
europepmc   +1 more source