Results 51 to 60 of about 392 (96)

Early Symptoms and Treatment Outcomes in Neuronal Ceroid Lipofuscinosis Type 2: Croatian Experience. [PDF]

J Pers Med
Radić Nišević J, Kolić I, Kostanjski M, Kovačević F, Prpić I.   +4 more
europepmc   +1 more source

First case of use of cerliponase alfa in a child with neuronal ceroid lipofuscinosis type 2 in Bahia

Arquivos de Neuro-Psiquiatria
Érica Otoni Pereira Miranda, Lukas Santos Freire, Giovana Andrade de Oliveira, Ícaro Giovani Barros Carregosa, Rebeca Lima de Almeida Santos, Qesya Rodrigues Ferreira, Odivan de Souza Lopes, Emmanuel Santos Trindade, Lorena Tanajura Oliveira   +8 more
openaire   +1 more source

PMDA Perspective on Use of Real-World Data and Real-World Evidence as an External Control: Recent Examples and Considerations. [PDF]

Clin Pharmacol Ther
Asano J, Sugano H, Murakami H, Noguchi A, Ando Y, Uyama Y.   +5 more
europepmc   +1 more source

Ceroid lipofuscinosis type 2 disease: Effective presymptomatic therapy-Oldest case of a presymptomatic enzyme therapy. [PDF]

Eur J Neurol
Breuillard D, Ouss L, Le Normand MT, Denis TS, Barnerias C, Robert MP, Eisermann M, Boddaert N, Caillaud C, Bahi-Buisson N, Desguerre I, Aubart M.   +11 more
europepmc   +1 more source

The expanding role of protease therapeutics (2012-2026): from replacement therapies to immune system modulation and beyond. [PDF]

Biochem J
Nelson SE, Martinusen SG, Pho R, Long LF, Denard CA.   +4 more
europepmc   +1 more source

OCT Biomarkers in Ocular CLN2 Disease in Patients Treated With Intraventricular Enzyme Replacement Therapy. [PDF]

Invest Ophthalmol Vis Sci
Huang WC, Ohnsman CM, Atiskova Y, Falabella P, Spitzer MS, Schulz A, Dulz S.   +6 more
europepmc   +1 more source

Peripapillary Retinal Nerve Fiber Layer (pRNFL) Thickness - A Novel Biomarker of Neurodegeneration in Late-Infantile CLN2 Disease. [PDF]

Eye Brain
Gkalapis N, Dulz S, Grohmann C, Nickel M, Schwering C, Wibbeler E, Spitzer MS, Schulz A, Atiskova Y.   +8 more
europepmc   +1 more source

Enzyme Replacement Therapy for CLN2 Disease: MRI Volumetry Shows Significantly Slower Volume Loss Compared with a Natural History Cohort. [PDF]

AJNR Am J Neuroradiol
Gaur P, Gissen P, Biswas A, Mankad K, Sudhakar S, D'Arco F, Schulz A, Fiehler J, Sedlacik J, Löbel U.   +9 more
europepmc   +1 more source

Access to Orphan Medicinal Products in Bulgaria: An Analysis of the Positive Drug List and Individual Access Schemes. [PDF]

Healthcare (Basel)
Stefanov R, Raycheva R, Kostadinov K, Stefanov G, Zdravkova-Aneva I, Dzhambazova E, Iskrov G.   +6 more
europepmc   +1 more source

Long-Term Data For Cerliponase Alfa ▼ (Brineura) In Children With CLN2 Disease: A Clinical Trial Update.

EMJ Neurology
openaire   +1 more source