Results 141 to 150 of about 1,989 (180)

Biomarkers of immune dysregulation and posttreatment inflammation in spinal muscular atrophy. [PDF]

Proc Natl Acad Sci U S A
Jordan SC, Bakhtiari M, Bhasin SS, Verma S, Bhasin MK.   +4 more
europepmc   +1 more source

Emerging Technologies Tackling Adeno-Associated Viruses (AAV) Immunogenicity in Gene Therapy Applications. [PDF]

Pharmaceutics
Egorova T, Starikova A, Polikarpova A.
europepmc   +1 more source

A type IV spinal muscular atrophy with gastrocnemius pseudohypertrophy caused by SMN1 deletion: a case report and literature review. [PDF]

BMC Neurol
Xi H, He W, Jiang H, Xie W, Yang Y, Yang Y, Diao K, Yang W, Li H.   +8 more
europepmc   +1 more source

SMN1 variants identified by false-positive SMA newborn screening tests: Therapeutic hurdles and functional and epidemiological solutions. [PDF]

Am J Hum Genet
Wirth B, Das J, Kölbel H, Goh S, Farrar MA, Piano V, Zetzsche S, Fuhrmann N, Becker J, Karakaya M, Zhang Y, Cao Y, Taghipour-Sheshdeh A, Stringer BW, Giacomotto J.   +14 more
europepmc   +1 more source

Strategies of AAV capsid engineering for targeted delivery to brain, muscle, and retina. [PDF]

Front Mol Biosci
Xu X.
europepmc   +1 more source

Gene therapy for spinal muscular atrophy: perspectives on the possibility of optimizing SMN1 delivery to correct all neurological and systemic perturbations. [PDF]

Neural Regen Res
Brown SJ, Yáñez-Muñoz RJ, Fuller HR, Fuller HR.   +3 more
europepmc   +1 more source

Engineered tumor specific AAV for IL-12 delivery in ovarian cancer immunotherapy. [PDF]

Acta Pharm Sin B
Tong F, Li H, Gao H, Li Y.
europepmc   +1 more source

The effectiveness and value of therapies for spinal muscular atrophy. [PDF]

J Manag Care Spec Pharm
Tice JA, Luu L, Sanchez S, Lee W, Nikitin D, Phillips M, Carlson JJ, Chan HH, Rind DM, Agboola F.   +9 more
europepmc   +1 more source

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Novartis Confirms Deaths of Two Patients Treated with Gene Therapy Zolgensma

Human Gene Therapy, 2022
exaly   +3 more sources

ZOLGENSMA-related fatal acute liver failure

Reactions Weekly, 2022
exaly   +2 more sources