Neurological and cardiac responses after treatment with miglustat and a ketogenic diet in a patient with Sandhoff disease.
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Less Is More: Substrate Reduction Therapy for Lysosomal Storage DisordersSubstrate reduction therapy for Krabbe's disease.Small molecules as therapeutic agents for inborn errors of metabolism.Emergencies in motoneuron disease.Protein misfolding diseases: prospects of pharmacological treatment.Clinical presentation and outcome in infantile Sandhoff disease: a case series of 25 patients from Iranian neurometabolic bioregistry with five novel mutations
P2860
Neurological and cardiac responses after treatment with miglustat and a ketogenic diet in a patient with Sandhoff disease.
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2014 nî lūn-bûn
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name
Neurological and cardiac respo ...... patient with Sandhoff disease.
@en
Neurological and cardiac respo ...... patient with Sandhoff disease.
@nl
type
label
Neurological and cardiac respo ...... patient with Sandhoff disease.
@en
Neurological and cardiac respo ...... patient with Sandhoff disease.
@nl
prefLabel
Neurological and cardiac respo ...... patient with Sandhoff disease.
@en
Neurological and cardiac respo ...... patient with Sandhoff disease.
@nl
P2093
P1476
Neurological and cardiac respo ...... patient with Sandhoff disease
@en
P2093
Gabriela Karl
Ives T Villamizar-Schiller
Laudy A Pabón
Norma C Serrano
Robert J Hopkin
Sophia B Hufnagel
P304
P356
10.1016/J.EJMG.2014.12.009
P577
2014-12-12T00:00:00Z