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Spinal Muscular Atrophy
Trials & Approvals Genentech
Genentech Unveils Positive Two-Year Data For Spinal Muscular Atrophy Drug
2022-10-13
Trials & Approvals AAV Gene Therapy
Novartis Reported Two Deaths Following Zolgensma Gene Therapy
2022-08-12
Trials & Approvals Spinal Muscular Atrophy
First Oral SMA Drug by Roche Now Available to Newborns
2022-06-01
Regulatory Gene Therapy
After FDA Lifts Hold, Novartis to Proceed with Clinical Trials for Expensive Rare Disease Therapy
2021-08-04
Trials & Approvals Gene Therapy
Novartis’ Expensive Gene Therapy for SMA Reasserts its Efficacy in Two Clinical Trials
2021-06-24
Technology
Jaguar Launches with a Pre-clinical Pipeline of AAV9-Based Gene Therapies
2021-02-28
R&D
Altering the Treatment Landscape of Neurodegenerative Diseases Using Antisense Oligonucleotides (ASOs)
2020-08-17
Trials & Approvals
Genentech Enters Spinal Muscular Atrophy Market with Risdiplam’s FDA Approval
2020-08-11
In-Depth
Spotlight: New Gene Therapies on the Horizon for the Treatment of Neurogenetic Diseases
2020-08-03
Trials & Approvals
Spotlight: Roche’s SMA Drug, Risdiplam Makes Strong Case for Regulatory Approval
2020-07-05
R&D
Biogen’s SMA Drug, Spinraza Continues to Show Benefits in Pre-Symptomatic Patients
2020-06-14
R&D
Weekly Cover: Eisai to Launch “NouKNOWTM” in Japan for Regular Self-assessment of Brain Health
2020-03-26
Trials & Approvals Rare Disease
FDA Okays Expensive Gene Therapy for Infantile-Onset Spinal Muscular Atrophy
2019-05-29
LATEST
AACR Annual Meeting 2025: Cancer Research Innovations, NIH Funding Advocacy, and AI-Driven Advances
2025-04-28
Senti Biosciences to Showcase New Cell & GeneTherapy Data at AACR Annual Meeting 2025
2025-04-27
World Vaccine Congress Washington 2025 Recap: Urgent Calls for Trust, Tech, and Global Access
2025-04-25
Astellas’ Transformation Journey: From Merger to Global Player, Betting on the Edge of Innovation and Risk?
2025-04-25
Roche Announces Massive 50 Billion Dollar Investment in the United States
2025-04-25
Steminent Stands Ready to Showcase Novel MSC-based Therapy for Spinocerebellar Ataxia at Global Stage
2025-04-24
Arkansas Medicaid Work Requirements Led to Coverage Loss for a Significant Number of Recipients
2025-04-24
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EVENT
2025-04-25
AACR Annual Meeting 2025
Chicago, U.S.A
2025-05-03
29th Taiwan Joint Cancer Conference 2025
Taipei, Taiwan
2025-05-05
Swiss Biotech Day 2025
Basel, Switzerland
2025-05-13
ASGCT 28th Annual Meeting 2025
New Orleans, U.S.A.
2025-05-30
ASCO Annual Meeting 2025
Chicago, U.S.A
2025-06-11
ISSCR 2025 Annual Meeting
Hong Kong
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