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
top of page
Highlights:
News Briefs
- Jan 18
- 3 min
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).
- Dec 12, 2023
- 3 min
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).
Patient Advocacy & Community Activism
- Mar 21
- 6 min
Beyond the Skin and Muscles: Understanding Dermatomyositis
Explore the complexities of living with dermatomyositis, a rare autoimmune disorder affecting both muscles and skin.
- Feb 16
- 7 min
From Struggle to Seeking Solutions: The Reality of Rare Disease Diagnostic Methods
From genetic testing to AI, explore the evolving landscape shaping the future of rare disease diagnosis.
- Feb 2
- 6 min
Navigating the Complexity of Danon Disease
Unveil the complexities of Danon Disease –a rare multisystem disorder. From genetics to management, explore the latest insights in this blog
Thought Leaders
- Jan 23
- 4 min
A Discussion with Michael Kornhauser - A Rare Disease Ally in Clinical Research
Discussing the impact of clinical studies on patients’ lives and Michael’s perspective as a clinical trials operations specialist.
- May 30, 2023
- 7 min
Thought Leadership in Mental Health For Rare Diseases
Discussing the impact of the rare disease lived experience on mental health and what is being done to address these evolving issues, with JC
bottom of page