Results 71 to 80 of about 69,398 (132)

PPARα and RXRα in the regulation of neuronal ceroid lipofuscinosis genes: implications for Batten disease therapy. [PDF]

NeuroImmune Pharm Ther
Chandra S, Pahan K.
europepmc   +1 more source

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

, 2017
T. Ajayi, H. Cahan, P. Slasor, N. Specchio, E. de los Reyes, D. Jacoby, P. Gissen, A. Schulz   +7 more
core   +1 more source

Rarely listed essential medicines in 158 national lists. [PDF]

J Pharm Policy Pract
Ul Haq MZ, Heredia C, Rizvi A, Workentin A, Persaud N.   +4 more
europepmc   +1 more source

Evidence of the impact of CLN2 and CLN3 Batten disease on families in the United Kingdom. [PDF]

Orphanet J Rare Dis
Mole SE, Gissen P, Nordstrom S, Wait S, Allen L, Antonini M, Brownnutt L, Brown R, Cole B, Gibbon F, Henderson RH, Kenrick S, Sisic Z, Thompson B, Nightingale J.   +14 more
europepmc   +1 more source

Long-term safety and efficacy of intracerebroventricular enzyme replacement therapy with cerliponase alfa in children with CLN2 disease: Two year results from an ongoing multicenter extension study

, 2018
Nicola Specchio, Peter Slasor, Temitayo Ajayi, David Jacoby, Paul Gissen, Angela Schulz, Heather Cahan, Emily de los Reyes   +7 more
core   +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

CLN2 disease (neuronal ceroid lipofuscinosis type 2): Experience in the real world with cerliponase alfa intracerebroventricular enzyme replacement therapy in a public hospital in Cordoba, Argentina

, 2018
Francisco Pueyrredon, Roberto Caraballo, Victor Munoz, Raul Jalil, Tatiana Rodrigo, Norberto Guelbert, Guillermo Seratti, Guillermo Guelbert, Daniel Velazquez   +8 more
core   +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