Yahoo Finance8d
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 ...
BioWorld9d
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).
BioSpace26d
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, ...
Canada6mon
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 ...