Results 101 to 110 of about 2,159 (173)

Produtos de terapia avançada aprovados pela ANVISA

Com o advento da terapia gênica foi possível ter uma nova perspectiva sobre tratamentos de diversas doenças posto que o medicamento age em nível genético visando correção das anormalidades causadoras das enfermidades.
Fogaça, Natali Pereira
core   +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

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

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

Nanocarrier-Based Gene Delivery Systems: Mechanisms, Clinical Translation, and Future Perspectives. [PDF]

Biotechnol Bioeng
Naqvi RF, Ali M, Zuberi SA, Aamir N, Rafique S.   +4 more
europepmc   +1 more source

Differences in swallowing efficacy of disease modifying treatment between infants receiving pre-symptomatic and symptomatic administration. [PDF]

Orphanet J Rare Dis
McGrattan KE, Mohr AH, Miles A, Allen J, Ochura J, Hernandez K, Walsh K, Rao V, Stevens M, McGhee H, Nichols K, Turksi ME, Spoden A, Wilson I, Coker M, Leon-Astudillo C, Smith LS, Brandsema J, Farah H, Welc J, Levy D, Clements M, Duong T, Young SD, Schenck G, Richardson R, Karachunski P, Brown A, Brown A, Castro D, Darras BT, Graham RJ.   +31 more
europepmc   +1 more source

Costs of Treating Onasemnogene Abeparvovec-Xioi-Induced Liver Injury. [PDF]

Pharmacol Res Perspect
Belančić A, Raičević B, Stević I, Vitezić D, Janković SM.   +4 more
europepmc   +1 more source

How social pharmaceutical innovations are addressing problems of availability, accessibility and affordability of drugs for rare diseases. [PDF]

Orphanet J Rare Dis
Douglas CMW, Kleinhout-Vliek T, Hagendijk R, Rabeharisoa V, Boon W, Aith F, Oliveira CC, Grunebaum S, Moors E.   +8 more
europepmc   +1 more source

Türkiye’de SMA’lı Çocukların Tedavisi ve Yabancı Ülke Örnekleri

SMA hastalığı nadir görülen bir gen hastalığıdır. SMA için şu anda uygulanmakta olan bir ilaç tedavisi mevcuttur. Son dönemlerde yapılan bu ilaç tedavisine “Zolgensma” isimli yeni ve pahalı bir ilaç daha eklenmiştir.
Arslan Ertürk, Arzu
core  

Onasemnogene Abeparvovec (Zolgensma) Decreases Ventilator/Bi-Pap Support in 3 Infants with SMA 1

Annals of Case Reports, 2021
openaire   +1 more source