Results 91 to 100 of about 25,675 (116)

Oxytocin in infants with Prader-Willi syndrome to improve dysphagia and disease trajectory. [PDF]

open access: yesOrphanet J Rare Dis
Tauber M   +19 more
europepmc   +1 more source

Allopurinol Treatment Improves Cognitive Skills, Adaptive Behavior, and Biochemical Markers in Young Patients With Adenylosuccinate Lyase Deficiency. [PDF]

open access: yesJ Inherit Metab Dis
Rousselot-Pailley B   +12 more
europepmc   +1 more source

Beyond Trikafta: new models to assess tissue dependent rescue of N1303K-CFTR. [PDF]

open access: yesFront Pharmacol
Pranke I   +63 more
europepmc   +1 more source

Editorial: Biology of lysosome-related organelles. [PDF]

open access: yesFront Cell Dev Biol
Delevoye C, Barral DC, Setty SRG.
europepmc   +1 more source

Nutritional Management in Severe Methylmalonic and Propionic Acidemias: How Much Medical Food Is Too Much? [PDF]

open access: yesJ Inherit Metab Dis
Margoses D   +19 more
europepmc   +1 more source

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