The SPARKLE registry: protocol for an international prospective cohort study in patients with alpha-mannosidosis.

ABSTRACT:

Background

Alpha-mannosidosis is a lysosomal storage disorder caused by reduced enzymatic activity of alpha-mannosidase. SPARKLE is an alpha-mannosidosis registry intended to obtain long-term safety and effectiveness data on the use of velmanase alfa during routine clinical care in patients with alpha-mannosidosis. It is a post-approval commitment to European marketing authorization for Velmanase alfa (Lamzede^®), the first enzyme replacement therapy for the treatment of non-neurologic manifestations in patients with mild to moderate alpha-mannosidosis. In addition, SPARKLE will expand the current understanding of alpha-mannosidosis by collecting data on the clinical manifestations, progression, and natural history of the disease in treated and untreated patients, respect

SUBMITTER: Hennermann JB 

PROVIDER: S-EPMC7525940 | biostudies-literature | 2020 Sep

REPOSITORIES: biostudies-literature