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FDA Extends Priority Review Period for Kresladi/ RP-L 201 in Treating Severe Leukocyte Adhesion Deficiency-I (LAD-1)
Explore the FDA's extension of the priority review period for Kresladi, a groundbreaking gene therapy for severe LAD-1
The Rare360 Editorial Team
Feb 19, 20242 min read
Milestone in Gene Therapy: Casgevy Approved for Transfusion-Dependent Beta Thalassemia
Explore the FDA's groundbreaking approval of CASGEVY™ the treatment of transfusion-dependent beta-thalassemia (TDT).
The Rare360 Editorial Team
Jan 18, 20243 min read
New Horizon in PNH Treatment: FDA Approves First Oral Drug
Explore the latest in PNH treatment with the FDA's groundbreaking approval of iptacopan (Fabhalta).
The Rare360 Editorial Team
Dec 12, 20233 min read
New Hope for Pediatric Patients: Bosutinib's Approval in Treating Chronic Phase Ph+ CML
Learn about Ph+ CML, its phases, and the FDA's approval of bosutinib for pediatric patients with chronic myeloid leukemia.
The Rare360 Editorial Team
Nov 6, 20233 min read
Assessing Safety and Effectiveness of Damoctocog Alfa Pegol in Hemophilia A Patients
HEM-POWR study shows damoctocog alfa pegol's promise, reducing bleeding and improving joint health in hemophilia A patients.
The Rare360 Editorial Team
Nov 1, 20233 min read
Late-Onset Pompe Disease: New Clinical Insights Pave the Way for Enhanced Quality of Life
A groundbreaking study offers hope to the LOPD community by revealing crucial clinical markers that could transform diagnosis and treatment.
The Rare360 Editorial Team
Sep 21, 20232 min read
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