Friday, March 1, 2024

Prime Medicine Reports Full Year 2023 Financial Results and Provides Business Updat

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CAMBRIDGE, Mass., March 01, 2024 (GLOBE NEWSWIRE) -- Prime Medicine, Inc. (Nasdaq: PRME), a biotechnology company committed to delivering a ...

After pair of failures, PTC finds a path through FDA jungle for Friedreich ataxia med

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https://www.fiercebiotech.com. By Annalee Armstrong; Mar 1, 2024. In a fourth-quarter earnings update Thursday afternoon, PTC executives bri...

PTC Therapeutics Provides Corporate Update and Reports Fourth Quarter and Full Year 2023 Financial Results

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SOUTH PLAINFIELD, N.J., Feb. 29, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. PTC had a Type C meeting with the FDA in the first quarter of 2...
Monday, February 26, 2024

Voyager Therapeutics Announces Selection of Gene Therapy Development Candidate for Friedreich’s Ataxia in Collaboration with Neurocrine Biosciences

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Voyager Therapeutics Announces Selection of Gene Therapy Development Candidate for Friedreich’s Ataxia in Collaboration with Neurocrine Bios...
Sunday, February 25, 2024

Phenotypic variation of FXN compound heterozygotes in a Friedreich ataxia cohort

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Shen MM, Rummey C, Lynch DR. Phenotypic variation of FXN compound heterozygotes in a Friedreich ataxia cohort. Ann Clin Transl Neurol. 2024 ...
Thursday, February 22, 2024

Generation of two human induced pluripotent stem cell lines, IGIBi012-A and IGIBi013-A from Friedreich's ataxia (FRDA) patients with homozygous GAA repeat expansion in FXN gene

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Ahmad I, Kapoor H, Srivastava AK, Faruq M. Generation of two human induced pluripotent stem cell lines, IGIBi012-A and IGIBi013-A from Fried...
Wednesday, February 21, 2024

Union Register of medicinal products for human use: (2024)975 of 09 Feb 2024 Skyclarys

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  https://ec.europa.eu/health/documents/community-register/2024/20240209161576/dec_161576_es.pdf https://ec.europa.eu/health/documents/commu...
Thursday, February 15, 2024

Larimar Therapeutics Reports Positive Top-line Data from Phase 2 Dose Exploration Study from 25 mg and 50 mg Cohorts of Nomlabofusp in Patients with Friedreich’s Ataxia

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BALA CYNWYD, Pa., Feb. 12, 2024 (GLOBE NEWSWIRE) -- Larimar Therapeutics, Inc. (“Larimar”) (Nasdaq: LRMR), a clinical-stage biotechnology co...
Monday, February 12, 2024

Biogen Received European Commission Approval for SKYCLARYS® (omaveloxolone), the First Therapy to Treat Friedreich’s Ataxia

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CAMBRIDGE, Mass., Feb. 12, 2024 (GLOBE NEWSWIRE) -- Biogen Inc. (Nasdaq: BIIB) announced the European Commission (EC) has authorized SKYCLA...
Friday, February 9, 2024

Real-world evidence for coverage determination of treatments for rare diseases

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Dayer, V.W., Drummond, M.F., Dabbous, O. et al. Real-world evidence for coverage determination of treatments for rare diseases. Orphanet J R...