Results 111 to 120 of about 2,159 (173)

Safety of Adeno-Associated Viral Vectors in Gene Therapy: Mechanisms of Toxicity, Clinical Risks, and Strategies for Their Minimization. [PDF]

Int J Mol Sci
Tsaregorodtseva TS, Radyukhina MA, Ayupova AI, Solovyeva VV, Sufianov AA, Sufianova GZ, Rizvanov AA.   +6 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

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

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

Parfitian Priority, Gene Therapy, and Disability. [PDF]

J Med Philos
Wilkinson D.
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

FRAME: Framework for Real-World Evidence Assessment to Mitigate Evidence Uncertainties for Efficacy/Effectiveness - An Evaluation of Regulatory and Health Technology Assessment Decision Making. [PDF]

Clin Pharmacol Ther
Candore G, Martin C, Mills MJ, Suter A, Lloyd A, Chavez-Montoya D, Civitelli D, Wolf B, Bolot P, Wasem J, Soriano Gabarró M, Kanavos PG, Sculpher M.   +12 more
europepmc   +1 more source

Immune Toxicities in AAV Gene Therapy: Overview for Clinicians. [PDF]

Int J Mol Sci
Parajuli S, Gallagher T, Flotte TR.
europepmc   +1 more source

DNA damage/p53, innate immune, and unfolded protein responses are activated in primate liver after toxic, high-dose AAV-SMN1 delivery. [PDF]

Mol Ther Adv
Moeini P, Bilbao-Arribas M, Guruceaga E, Torrens-Baile J, Lanz TA, Whiteley LO, Aragón T, Unzu C, González-Aseguinolaza G.   +8 more
europepmc   +1 more source

Current State-Of-Play of the EU Advanced Therapy Medicinal Product (ATMP) Field, With an Emphasis on Belgian Human Cell and Tissue Products. [PDF]

Wound Repair Regen
Verbeken G, Convents L, Delmotte N, Draye JP, Jennes S, Vanderkelen A, Nijs G, Lewalle P, Baudoux E, Cornu O, Vanlaere I, Pierlot A, Rose T, Pirnay JP.   +13 more
europepmc   +1 more source