Alnylam Pharmaceuticals Reports Fourth Quarter and Full Year 2024 Financial Results and Highlights Recent Period Progress
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today reported its consolidated financial results for the fourth quarter and ...
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FDA grants orphan drug status to Arbor Biotechnologies’ PH1
The US Food and Drug Administration (FDA) has granted orphan drug designation (ODD) and rare paediatric disease designation ...
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Arbor’s ABO-101 awarded US orphan drug designation for primary hyperoxaluria type 1
Arbor Biotechnologies Inc.’s ABO-101 has been awarded orphan drug and rare pediatric disease designations by the FDA for the treatment of primary hyperoxaluria type 1 (PH1).
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Government of Canada signs bilateral agreement with Ontario for Drugs for Rare Diseases
for the treatment of hyperoxaluria type 1; Epkinly, for relapsed or refractory diffuse large B-cell lymphoma; Welireg, for the treatment of treatment of von Hippel-Lindau (VHL) disease; and, Yescarta, ...
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YolTech Therapeutics to Initiate a Clinical Trial for YOLT-204, a First-in-Class Bone Marrow-Targeted In Vivo Gene Editing Therapy for β-Thalassemia
SHANGHAI, Jan. 20, 2025 /PRNewswire/ -- YolTech Therapeutics, a clinical-stage gene editing company dedicated to delivering lifelong cures, announced the initiation of a clinical trial for YOLT-204, ...
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Drugs for rare diseases: Common list of drugs
Poteligeo (Mycosis fungoides or Sézary syndrome) Oxlumo (Primary hyperoxaluria type 1) Epkinly (Relapsed or refractory diffuse large B-cell lymphoma) Welireg (Von Hippel-Lindau disease) Yescarta ...