hematopoietic stem cell transplantation
Feb 16, 2026
Alternative Therapeutic Strategies Beyond HSCT: Reimagining Curative Options in Hematologic Disease
Summary HSCT is a potentially curative treatment for hematologic malignancies (acute leukemias, lymphomas, multiple myeloma) and bone marrow failure syndromes. In 2025, approximately 26,000 HSCT cases were reported in the 7MM, with Multiple Myeloma/Plasma Cell Dyscrasias having the largest patient pool in th...
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Jan 05, 2026
FDA Approves Omeros’ YARTEMLEA: A Breakthrough for Stem Cell Transplant Patients Facing TA-TMA
The FDA has approved YARTEMLEA (narsoplimab-wuug), marking a watershed moment in hematopoietic stem cell transplantation (HSCT) medicine. Omeros Corporation's therapeutic advance represents the first and only FDA-approved treatment for hematopoietic stem cell transplant-associated thrombotic microangiopathy (TA-TMA...
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Dec 19, 2025
WASKYRA Wins FDA Approval, Marking a Historic Leap for Rare Disease Gene Therapies
In a landmark decision on December 10, 2025, the FDA approved WASKYRA (etuvetidigene autotemcel), marking a transformative milestone in the treatment of Wiskott-Aldrich syndrome—a rare, life-threatening genetic blood disorder. This approval represents not only a significant advancement in gene therapy but also a hi...
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Sep 12, 2023
BMS’s LPA1 Antagonist; Alnylam’s KARDIA-1 Phase 2 Study; Day One Biopharma Sought FDA Approval for Tovorafenib; EMA Orphan Drug Designation to MaaT Pharma’s MaaT033; Lundbeck and Otsuka Announce Topline Results from Two Phase III Trials of Brexpiprazole + Sertraline; Phase III CheckMate – 227 Trial Show Durable, Long-Term Survival with Opdivo Plus Yervoy
Bristol Myers Squibb’s Investigational LPA1 Antagonist Reduces Rate of Lung Function Decline in Progressive Pulmonary Fibrosis Cohort of Phase II Study BMS-986278, a potential first-in-class oral lysophosphatidic acid receptor 1 (LPA1) antagonist, was studied in patients with progressive pulmonary fibrosis (PPF)...
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May 24, 2022
PTC Therapeutics’ Gene Therapy Upstaza; Sanofi and Regeneron’s Dupixent; Bayer CAR-T Collaboration with Atara; FDA Accepts Biohaven’s Zavegepant; AbbVie Files FDA Approval for ABBV-951; Innoviva to Acquire Entasis; FDA Orphan Drug Designation to XMT-2056; FDA Approves Azacitidine for Juvenile Myelomonocytic Leukemia
EU Recommends Approval for PTC Therapeutics’ Gene Therapy Upstaza Upstaza, a gene therapy developed by PTC Therapeutics for patients with the genetic condition AADC deficiency, has been recommended for EU approval, putting another test of gene therapy's commercial prospects in the union. Upstaza (eladocagene exu...
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