Latest PNH News
Features & Insights
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.
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…
PNH has been described as the “great impersonator” due to its ability to present with nonspecific symptoms.
Rare disease advocates are marking the 40th anniversary of the landmark Orphan Drug Act, which has led to over 1100 approvals for new therapies.
The Bespoke Gene Therapy Consortium (BGTC) is an ambitious public-private partnership that aims to develop gene therapies for ultrarare diseases.
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