Results 61 to 70 of about 165,283 (121)

Dose selection for biological enzyme replacement therapy indicated for inborn errors of metabolism. [PDF]

Clin Transl Sci, 2023
Hon YY, Wang J, Abodakpi H, Balakrishnan A, Pacanowski M, Chakder S, Smpokou P, Donohue K, Wang YC.   +8 more
europepmc   +1 more source

Apresentação

, 2001
Alfa, Revista
core   +4 more sources

Neuronal ceroid lipofuscinosis: underlying mechanisms and emerging therapeutic targets. [PDF]

Nat Rev Neurol
Ziółkowska EA, Takahashi K, Dickson PI, Sardiello M, Sands MS, Cooper JD.   +5 more
europepmc   +1 more source

1973. Domenico Rea a Alfa Romeo - Alfa sud

, 1973
Rea, Domenico, Alfa Romeo - Alfasud
core   +1 more source

Intravitreal Enzyme Replacement Therapy Slows Retinopathy in Late Infantile Ceroid Lipofuscinosis Type 2. [PDF]

Neuropediatrics
Priglinger CS, Courage C, Lotz-Havla AS, Gerhardt M, Ehrt O, Kurz M, Pudritz H, Rudolph G, Jackson CB, Maier EM.   +9 more
europepmc   +1 more source

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

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

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

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