Latest PNH News
Ravulizumab Offers Benefits in PNH, but Hemolysis Still Possible
Study Suggests Model for Mitigating DTDCs in PNH Patients Who Switch to Crovalimab
Case Study: Paroxysmal Nocturnal Hemoglobinuria Concurrent With Aplastic Anemia
Ravulizumab Offers Less Frequent Infusions and Improved Quality of Life in PNH
CFH Variants Over-Represented in PNH May Influence Treatment Response
Oral Factor D Inhibitor Achieves Positive Results in Phase 1 Complement System Trial
Long Noncoding RNA FAM157C May Boost Clonal Proliferation in PNH
New Insights Into IFN-α Signaling in Paroxysmal Nocturnal Hemoglobinuria
Positive Results Seen With NM3086 Treatment in PNH Animal Model
Features & Insights
Omidubicel Shows Promise as Replacement for Umbilical Cord Blood in Stem Cell Transplants
Gamida Cell is gearing up to begin sales of omidubicel from Israel to US hospitals for use in stem cell transplants, pending FDA approval of the therapy.
The Dangerous Implications of Paroxysmal Nocturnal Hemoglobinuria
Paroxysmal nocturnal hemoglobinuria (PNH) is a condition that is poorly defined and difficult to diagnose. Characterized by episodic complement-mediated hemolysis, its classic presentation is the sudden, episodic, and night-time occurrence of hemoglobin in the urine. The most common symptom shared by patients with this disease is debilitating fatigue. According to the International Paroxysmal Nocturnal Hemoglobinuria…
Paroxysmal Nocturnal Hemoglobinuria: The Great Impersonator
PNH has been described as the “great impersonator” due to its ability to present with nonspecific symptoms.
At 40, Orphan Drug Act Enjoys Rare Bipartisan Support
Rare disease advocates are marking the 40th anniversary of the landmark Orphan Drug Act, which has led to over 1100 approvals for new therapies.
Ambitious NCATS Bespoke Gene Therapy Consortium Targets Ultrarare Diseases
The Bespoke Gene Therapy Consortium (BGTC) is an ambitious public-private partnership that aims to develop gene therapies for ultrarare diseases.
PNH Clinical Trials
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