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January 30, 2025 07:37 ET | Source: uniQure Inc.

uniQure Announces Favorable Recommendation from Independent Data Monitoring Committee for its Phase I/II EPISOD1 Clinical Trial of AMT-162 for the Treatment of SOD1-ALS

~ Independent Data Monitoring Committee Recommends Proceeding with Dose Escalation After Planned Safety Assessment of First Dose Cohort ~ ~ Company Expects to Initiate Enrollment of Second Dose...
October 15, 2024 07:05 ET | Source: uniQure Inc.

uniQure Announces Dosing of First Patient in Phase I/II Clinical Trial of AMT-162 for the Treatment of SOD1-ALS

LEXINGTON, Mass. and AMSTERDAM, Oct. 15, 2024 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical...
January 31, 2023 07:05 ET | Source: uniQure Inc.

uniQure and Apic Bio enter into global licensing agreement for APB-102, a clinical stage gene therapy for patients with ALS caused by mutations in SOD1

~ License of APB-102 further strengthens uniQure’s pipeline of innovative gene therapies to treat neurological disorders and miRNA-based gene silencing programs ~ ~ APB-102 and uniQure’s c9orf72-ALS...
September 21, 2022 17:15 ET | Source: Biogen Inc.

The New England Journal of Medicine Publishes Pivotal Tofersen Data that Show Benefits in Rare, Genetic Form of ALS

Publication includes the Phase 3 VALOR SOD1-ALS trial and its open-label-extension study, underscoring the importance of these longer-term data12-month data show earlier initiation of tofersen slowed...
May 01, 2019 16:30 ET | Source: Biogen Inc.

Biogen to Present New Interim Data from Its Phase 1/2 Clinical Study of Tofersen (BIIB067) for the Potential Treatment of a Subtype of Familial Amyotrophic Lateral Sclerosis (ALS)

Tofersen, an antisense oligonucleotide (ASO) that selectively targets the genetic driver of disease, is being studied for the potential treatment of ALS in adults with a confirmed superoxide dismutase...
December 06, 2018 07:30 ET | Source: Biogen Inc.; Ionis Pharmaceuticals, Inc.

Biogen Exercises Option with Ionis to Develop and Commercialize Investigational Treatment BIIB067 for a Subtype of Familial Amyotrophic Lateral Sclerosis (ALS) Based on Positive Phase 1 Data

Positive interim analysis demonstrated proof-of-biology and proof-of-concept for BIIB067Biogen plans to advance BIIB067 to a pivotal clinical studyBiogen paid Ionis Pharmaceuticals a $35 million...