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PTC Therapeutics Announces Topline Results from Vatiquinone MOVE-FA Registration-Directed Trial - Vimarsana News

PTC Therapeutics Announces Topline Results from Vatiquinone MOVE-FA Registration-Directed Trial

- Conference call and webcast to be held at 5:00 pm EDT -  SOUTH PLAINFIELD, N.J., May 23, 2023 /PRNewswire/ -- PTC Therapeutics, Inc. today reported topline results from the MOVE-FA... | May 23, 2023

Acute myeloid leukaemia - Vimarsana News

Acute myeloid leukaemia

Progress in acute myeloid leukaemia treatment is occurring at an unprecedented pace. The past decade has witnessed an increasingly improved scientific understanding of the underlying biology of acute myeloid leukaemia, leading to enhanced prognostication tools and refined risk assessments, and most especially incorporating measurable residual disease (MRD) into longitudinal risk assessments. The classification of acute myeloid leukaemia has recently been updated by WHO and the International Consensus Classification (ICC).

Idorsia Pharmaceuticals Ltd: Idorsia to further characterize lucerastat for the treatment of Fabry disease by continuing the open-label extension of the Phase 3 MODIFY study - Vimarsana News

Idorsia Pharmaceuticals Ltd: Idorsia to further characterize lucerastat for the treatment of Fabry disease by continuing the open-label extension of the Phase 3 MODIFY study

Ad hoc announcement pursuant to Art. 53 LR Idorsia to further characterize lucerastat for the treatment of Fabry disease by continuing the open-label extension of the Phase 3 MODIFY study Idorsia

CHOP Researchers Find Effective Combination of Therapies for Managing Mitochondrial Disease - Vimarsana News

CHOP Researchers Find Effective Combination of Therapies for Managing Mitochondrial Disease

CHOP Researchers Find Effective Combination of Therapies for Managing Mitochondrial Disease Study is first to demonstrate how one specific combinational therapy may provide measurable benefits to patients with mitochondrial respiratory chain disorders, which currently lack FDA-approved therapies Newswise — Philadelphia, March 2, 2021 – Researchers from the Mitochondrial Medicine Frontier Program at Children’s Hospital of Philadelphia (CHOP) have demonstrated how one combination of therapies may be beneficial for patients with mitochondrial respiratory chain disorders. This preclinical r...

Frontiers | Barriers and Considerations for Diagnosing Rare Diseases in Indigenous Populations - Vimarsana News

Frontiers | Barriers and Considerations for Diagnosing Rare Diseases in Indigenous Populations

* 2National Centre for Indigenous Genomics, Australian National University, Canberra, ACT, Australia 3Department of Pharmacology, Dalhousie University, Halifax, NS, Canada 4Precision Medicine, Platform on Shaping the Future of Health and Healthcare, World Economic Forum, San Francisco, CA, United States 5Institute of Genetics, Canadian Institutes of Health Research, Government of Canada, Ottawa, ON, Canada 6Human Metabolomics, North-West University, Potchefstroom, South Africa 7Facultad de Medicina, Center for Genetics and Genomics, Clinica Alemana Universidad del Desarrollo, Santiago, Chile ...