Ambroxol chaperone therapy for neuronopathic Gaucher disease: A pilot study

Annals of Clinical and Translational Neurology

Volumn 3, Issue 3, 2016, Pages 200-215

  Narita, Aya ^a   Shirai, Kentarou ^a   Itamura, Shinji ^a   Matsuda, Atsue ^a   Ishihara, Akiko ^b   Matsushita, Kumi ^b   Fukuda, Chisako ^a   Kubota, Norika ^c   Takayama, Rumiko ^d   Shigematsu, Hideo ^d   Hayashi, Anri ^e   Kumada, Tomohiro ^e   Yuge, Kotaro ^f   Watanabe, Yoriko ^f   Kosugi, Saori ^g   Nishida, Hiroshi ^g   Kimura, Yukiko ^g   Endo, Yusuke ^b   Higaki, Katsumi ^h   Nanba, Eiji ^h   Nishimura, Yoko ^a   Tamasaki, Akiko ^a   Togawa, Masami ^a   Saito, Yoshiaki ^a   Maegaki, Yoshihiro ^a   Ohno, Kousaku ^a   Suzuki, Yoshiyuki ⁱ   more..

^a TOTTORI UNIVERSITY   (Japan)

^b TOTTORI UNIVERSITY HOSPITAL   (Japan)

^c National Hospital Organization Matsue National Hospital 5 8 31   (Japan)

^d SHIZUOKA INSTITUTE OF EPILEPSY AND NEUROLOGICAL DISORDERS   (Japan)

^e Medical Center for Children Shiga   (Japan)

^f KURUME UNIVERSITY SCHOOL OF MEDICINE   (Japan)

^g SHIN NIPPON BIOMEDICAL LABORATORIES LTD   (Japan)

^h TOTTORI UNIVERSITY   (Japan)

ⁱ TOKYO METROPOLITAN INSTITUTE OF MEDICAL SCIENCE   (Japan)

Author keywords

[No Author keywords available]

Indexed keywords

AMBROXOL; CHAPERONE; DIPEPTIDYL CARBOXYPEPTIDASE; GLUCOSYLCERAMIDASE; GLUCOSYLSPHINGOSINE; HEMOGLOBIN; SPHINGOSINE; UNCLASSIFIED DRUG; URIC ACID;

ADULT; ARTICLE; BLOOD BRAIN BARRIER; BLOOD SAMPLING; CEREBROSPINAL FLUID; CLINICAL ARTICLE; CLINICAL TRIAL; DRUG MEGADOSE; DRUG SAFETY; DRUG TOLERABILITY; DYSTONIA; ELECTROOCULOGRAPHY; ENZYME ACTIVITY; ENZYME REPLACEMENT; FEMALE; FUNCTIONAL INDEPENDENCE MEASURE; GAUCHER DISEASE; GROSS MOTOR FUNCTION MEASURE; HUMAN; LATENT PERIOD; LIMIT OF QUANTITATION; LIQUID CHROMATOGRAPHY; LUMBAR PUNCTURE; LYMPHOCYTE; MOTOR DYSFUNCTION ASSESSMENT; MOTOR PERFORMANCE; MUSCLE TONE; MYOCLONUS SEIZURE; OPEN STUDY; PRIORITY JOURNAL; PUPIL REFLEX; QTC INTERVAL; SACCADIC EYE MOVEMENT; TANDEM MASS SPECTROMETRY; TONIC CLONIC SEIZURE; UNIFIED MYOCLONUS RATING SCALE;

EID: 85009973979     PISSN: None     EISSN: 23289503     Source Type: Journal    
DOI: 10.1002/acn3.292     Document Type: Article

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