Results 111 to 120 of about 2,204 (179)

Impact of Intermittent Positive Pressure Breathing on Thoracic Growth in Children With Type 1 Spinal Muscular Atrophy. [PDF]

Pediatr Pulmonol
Thébault C, Denamur S, Thomas G, Barzic A, Schwartz E, Dupuis M, Nougues MC, Isapof A, Aubertin G, Taytard J.   +9 more
europepmc   +1 more source

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

Annals of Case Reports, 2021
openaire   +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

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

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

Correction to "Confidential and non-confidential drug rebates in Switzerland, 2013-2023: empirical analysis of drug rebates, trends over time and re-evaluations" [The Lancet Regional Health-Europe, volume 46, November 2024, 101094]. [PDF]

Lancet Reg Health Eur
Rohner N, Herrle C, Locher L, Vokinger KN.   +3 more
europepmc   +1 more source

Joint Scientific Consultation Eligibility Criterion: Hubris or Naïveté. [PDF]

J Mark Access Health Policy
Toumi M, Falissard B, Boyer L, Auquier P.   +3 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

Course of joint range of motion in children with spinal muscular atrophy receiving disease-modifying treatment. [PDF]

Orphanet J Rare Dis
Oude Lansink ILB, Gorter JW, van der Pol WL, Vd Woude DR, Scheiberlich PJ, van Eijk RPA, Bartels B, Beelen A.   +7 more
europepmc   +1 more source

Gene Therapy in Rare Genetic Disorders: Current Progress and Future Perspectives. [PDF]

Curr Genomics
Khawaja S, Ali RH, Ahmed I, Umair M.
europepmc   +1 more source