نتائج البحث

Real-World Outcomes in Patients with Spinal Muscular Atrophy Treated with Onasemnogene Abeparvovec Monotherapy: Findings from the RESTORE Registry.

• Authors : Servais, Laurent; Day, John W; De Vivo, Darryl C

Subjects: AAV9 vector-based gene replacement therapy; RESTORE registry; long-term follow-up

• Source: Journal of Neuromuscular Diseases, 11 (2), 425 - 442 (2024)

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Gene Therapy in ALS and SMA: Advances, Challenges and Perspectives.

• Authors : Lejman J; Student Scientific Society, Independent Laboratory of Genetic Diagnostics, Medical University of Lublin, 20-093 Lublin, Poland.; Panuciak K

Subjects: Amyotrophic Lateral Sclerosis*/Amyotrophic Lateral Sclerosis*/Amyotrophic Lateral Sclerosis*/genetics ; Amyotrophic Lateral Sclerosis*/Amyotrophic Lateral Sclerosis*/Amyotrophic Lateral Sclerosis*/therapy ; Muscular Atrophy, Spinal*/Muscular Atrophy, Spinal*/Muscular Atrophy, Spinal*/genetics

• Source: International journal of molecular sciences [Int J Mol Sci] 2023 Jan 06; Vol. 24 (2). Date of Electronic Publication: 2023 Jan 06.Publisher: MDPI Country of Publication: Switzerland NLM ID: 101092791 Publication Model: Electronic Cited Medium: Internet ISSN: 1422-0067

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Onasemnogene Abeparvovec (AVXS-101) for the Treatment of Spinal Muscular Atrophy.

• Authors : Naveed, Aimen¹ ; Calderon, Hillary²

• Source: Journal of Pediatric Pharmacology & Therapeutics. 2021, Vol. 26 Issue 5, p437-444. 8p.

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Gene Therapy in ALS and SMA: Advances, Challenges and Perspectives

• Authors : Jan Lejman; Kinga Panuciak; Emilia Nowicka

Subjects: gene therapy; motor neuron disease; SMAagris

• Source: International Journal of Molecular Sciences; Volume 24; Issue 2; Pages: 1130

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Combinatorial treatment for spinal muscular atrophy

• Authors : Poletti, Angelo; Fischbeck, Kenneth H.;

Subjects: Spinal muscular atrophy; motor neuron disease; therapy

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