Neuronal Ceroid Lipofuscinoses
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EMA approval · 2026-06-11EMA Authorised: Brineura (cerliponase alfa) — BioMarin International Limited
EMA confirms authorisation of Brineura (cerliponase alfa) for Neuronal Ceroid-Lipofuscinoses, designated as an orphan medicine with accelera…
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CLINICAL_TRIALS research · 2026-04-08Clinical Trial Update: Longitudinal Assessment of Atypical Tripeptidyl Peptidase 1 Enzyme Deficiency Patients
An update on a longitudinal assessment study of patients with Tripeptidyl peptidase 1 enzyme deficiency, including CLN2 disease and related …
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CLINICAL_TRIALS research · 2026-03-30Clinical Trial Update: Caregiving Networks Across Disease Context and the Life Course
The National Human Genome Research Institute sponsors a study examining caregiving networks across disease contexts and life stages, current…
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EMA approval · 2023-12-12EMA Authorised: Brineura (cerliponase alfa) — BioMarin International Limited
Brineura (cerliponase alfa) has been authorised by the EMA for Neuronal Ceroid-Lipofuscinoses, with orphan medicine status and accelerated a…