Biomarin Pharmaceutical Inc.

Charles A O'Neill

Augustus O Okhamafe

Saeed Moshashaee

Thomas Lester

Jason K Pinkstaff

Todd M Oppeneer

Thomas W Lester

The disclosure relates to the use of variants of C-type natriuretic peptide (CNP) to treat osteoarthritis, to ameliorate one or more symptoms of osteoarthritis, and to treat disorders having an osteoarthritis component.

Use of c-type natriuretic peptide variants to treat osteoarthritis

Formulations comprising recombinant human tripeptidyl peptidase-1 (rhTPP1) for intrathecal, intracerebroventricular, or intraocular administration, and kits comprising the same, are disclosed. Methods of using rhTPP1 in the prevention and treatment of symptoms of Neuronal Ceroid Lipofuscinosis (CLN2) disease are also disclosed. The formulations and methods are effective in halting the progression of CLN2 disease and may be used to treat subjects having CLN2 or a family history of CLN2.

TPP-1 formulations and methods for treating CLN2 disease

Use of C-type natriuretic peptide variants to treat osteoarthritis

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Vineburg, CA, United States of America

Charles A O'Neill