Results 111 to 120 of about 165,283 (121)

Seizures and Movement Disorders in Patients with CLN2 Disease Treated with Cerliponase Alfa in the Real-World Setting

Neuropediatrics
Angela Schulz, Miriam Nickel, Christoph Schwering, Eva Wibbeler, Lena Marie Westermann, Luca Hagenah, Sheila Reddy, Abigail Hunt, Matthias Hunger, Olivia Okoli, Pascal Reisewitz   +10 more
openaire   +1 more source

Brainstem auditory evoked potentials (BAEP) in CLN2 patients under treatment with cerliponase alfa

Molecular Genetics and Metabolism
Christoph Schwering, Angela Schulz, Christian Posern   +2 more
openaire   +1 more source

New product intros (brigatinib, cerliponase alfa, niraparib hydrochloride, midostaurin, ocrelizumab, valbenazine)

Drugs of Today, 2017
openaire   +1 more source

Intracerebroventricular cerliponase alfa for children with CLN2 disease: Interim results from an ongoing phase 2 extension study

European Journal of Paediatric Neurology, 2017
A. Schulz, N. Specchio, P. Gissen, E. de los Reyes, H. Cahan, P. Slasor, T. Ajayi, D. Jacoby   +7 more
openaire   +1 more source

Cerliponase alfa for the treatment of CLN2 disease in a patient cohort including children under 3 years of age

Molecular Genetics and Metabolism
Angela Schulz, Emily C. de los Reyes, Nicola Specchio, Paul Gissen, Peter Slasor, Shailesh Bondade, Jessica L. Cohen Pfeffer   +6 more
openaire   +1 more source

Presymptomatic and symptomatic disease stage-dependent outcomes of cerliponase alfa treatment in siblings with CLN2 disease

Molecular Genetics and Metabolism
Lena Marie Westermann, Lara Y. Ruhberg, Susanne Lezius, Luca Hagenah, Eva Heemann, Miriam Nickel, Christoph Schwering, Christian Posern, Angela Schulz   +8 more
openaire   +1 more source

Serial brain MRI volumetrics and DTI analysis in atypical CLN2 patients treated with ICV cerliponase alfa

Molecular Genetics and Metabolism
Sumiko Abe, Harriet Chang, Audrey Kao, Maija Steenari, Raymond Y. Wang   +4 more
openaire   +1 more source

Cerliponase alfa and neuronal ceroid lipofuscinosis type 2: long-term outcomes and lessons for future research

The Lancet Neurology
openaire   +2 more sources

A long-term observation of ophthalmology assessment in atypical CLN2 patients receiving ICV cerliponase alfa treatment

Molecular Genetics and Metabolism
Audrey Kao, Harriet Chang, Rahul Bhola, Raymond Y. Wang   +3 more
openaire   +1 more source

Serial brain MRI volumetrics and tractography of atypical CLN2 patients receiving ICV cerliponase alfa treatment

Molecular Genetics and Metabolism
Audrey Kao, Harriet Chang, Igor Nestrasil, Raymond Y. Wang   +3 more
openaire   +1 more source