Results 91 to 100 of about 4,199 (162)

Longitudinal deep multi-omics profiling in a CLN3<sup>Δex7/8</sup> minipig model identifies biomarker signatures of disease. [PDF]

open access: yesCommun Med (Lond)
Rechtzigel MJ   +11 more
europepmc   +1 more source

Therapeutic antisense oligonucleotide mitigates retinal dysfunction in a pig model of CLN3 Batten disease. [PDF]

open access: yesNucleic Acids Res
Stratton MP   +15 more
europepmc   +1 more source

Long-Term Open-Label Study Evaluating Oral Miglustat Treatment in Patients With Neuronal Ceroid Lipofuscinosis Type 3. [PDF]

open access: yesNeurology
Pietrafusa N   +9 more
europepmc   +1 more source

Visual Recovery and Neurological Stabilization Following Miglustat Treatment in Pediatric CLN3 Disease. [PDF]

open access: yesJ Child Neurol
Dutton AE   +6 more
europepmc   +1 more source

CLN3 disease disrupts very early postnatal hippocampal maturation. [PDF]

open access: yesSci Rep
Singh JB   +8 more
europepmc   +1 more source

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