Tag: Rare Diseases

Abu Dhabi Activates Rare Diseases Centre of Excellence

The Department of Health โ€“ Abu Dhabi, AstraZeneca, and PureHealth have partnered…

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Abu Dhabi Health Authority and PHRMAG Unite to Boost Oncology and Rare Disease Access

Ma'an and PHRMAG have partnered to improve access to oncology and rare…

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Abu Dhabi enhances International life sciences cooperation and investment opportunities

Abu Dhabiโ€™s Department of Health has partnered with Biocom California to establish…

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Novel Drug Therapy Offers Hope for Children With Brain-Injury-Related Obesity, Study Finds

Sidra Medicine has published a groundbreaking case report highlighting the successful use…

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Vertex Presents New CASGEVY Data in Children, Expands Global Regulatory Filings

Vertex Pharmaceuticals presented new data showing positive outcomes with CASGEVY in children…

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Sanofi provides update on MOBILIZE phase 3 study of riliprubart in chronic inflammatory demyelinating polyneuropathy

Sanofi has announced the discontinuation of its Phase 3 MOBILIZE study evaluating…

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Nivey Nocher Joins The PHA Group to Lead Health Department

The PHA Group has appointed Nivey Nocher as Executive Vice President, Health,…

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Pfizer Reaches Three Settlement Agreements for VYNDAMAX

Pfizer has reached settlement agreements with multiple generic manufacturers over VYNDAMAX (tafamidis),…

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Advancing Sickle Cell Cure in Africa: Transplant & Gene Therapy | Dr. Lawrence Faulkner | BMT

Advances in transplant medicine and gene editing are reshaping the future of…

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KFSH Successfully Implements Gene Therapy for Sickle Cell Disease

King Faisal Specialist Hospital & Research Centre (KFSH) successfully implements gene therapy…

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