In draft guidance, the National Institute for Health and Care Excellence has announced that the size and nature of any...
Chiesi Global Rare Diseases, a business unit of Chiesi Farmaceutici announced that the FDA has approved Lamzede (velmanase alfa-tycv) for the treatment of non-central nervous system manifestations of alpha-mannosidosis (AM) in adult and pediatric patients. AM is an ultra-rare, progressive lysosomal storage disorder caused by deficiency in the enzyme alpha-mannosidase.
The Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion, recommending the granting of a marketing authorisation...
Enzyme replacement therapy for the treatment of non-neurological manifestations in patients with mild to moderate alpha-mannosidosis. See sections 4.4 and 5.1.