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 […]

ikrisuser
January 14, 2022