Tag Archives: Rare disease
First and Only Enzyme Replacement Therapy Approved for Rare Alpha-Mannosidosis
The US FDA has approved Lamzede (velmanase alfa) for the treatment of non-central nervous system manifestations of alpha-mannosidosis (AM) in […]
ikrisuser
March 14, 2023
Rare Disease Medicines: India
What are rare diseases? A rare disease is a disease that affects a small population of the world or few […]
ikrisuser
March 14, 2023
What is NOMID and Its Treatment
NOMID is the most severe form of Cryopyrin-Associated Periodic Syndromes (CAPS), which are a group of autoinflammatory disorders seen in […]
ikrisuser
March 5, 2023
All you need to know about Cystinosis
Globally, more than 2,000 people have cystinosis, which is a rare, multisystem genetic disease or disorder. Cystinosis is characterized by […]
