|1.||Platt, Frances M: 13 articles (01/2016 - 08/2003)|
|2.||Butters, Terry D: 9 articles (08/2013 - 08/2004)|
|3.||Dwek, Raymond A: 9 articles (10/2008 - 05/2002)|
|4.||Bembi, Bruno: 8 articles (03/2015 - 03/2007)|
|5.||Giraldo, Pilar: 8 articles (08/2013 - 09/2005)|
|6.||Pastores, Gregory M: 6 articles (10/2011 - 02/2003)|
|7.||Abel, Larry: 5 articles (09/2015 - 09/2007)|
|8.||Patterson, Marc C: 5 articles (01/2015 - 09/2007)|
|9.||Vanier, Marie T: 5 articles (01/2015 - 02/2009)|
|10.||Zimran, Ari: 5 articles (08/2013 - 03/2002)|
|1.||Gaucher Disease (Gaucher's Disease)
03/01/2002 - "We conclude that OGT 918 was safe and effective at 50 mg TID, but shows dose dependency in ameliorating parameters of Gaucher disease relative to the results noted in the seminal trial; there was no improvement in the rate of hematological response and no reduction in side effects. "
02/01/2003 - "Substrate reduction therapy: miglustat as a remedy for symptomatic patients with Gaucher disease type 1."
04/01/2008 - "This review article discusses the results of clinical studies and use of miglustat as a therapeutic agent in patients with type I Gaucher disease."
09/01/2007 - "The safety and tolerability of miglustat 200 mg three times a day in study participants was consistent with previous trials in type I Gaucher disease, where half this dose was used. "
08/01/2005 - "An open-label, noncomparative study of miglustat in type I Gaucher disease: efficacy and tolerability over 24 months of treatment."
|2.||Type C Niemann-Pick Disease (Niemann Pick Disease, Type C)
01/01/2015 - "Long term follow-up to evaluate the efficacy of miglustat treatment in Italian patients with Niemann-Pick disease type C."
01/01/2013 - "Long-term efficacy of miglustat in paediatric patients with Niemann-Pick disease type C."
09/01/2015 - "Longitudinal changes in cerebellar and subcortical volumes in adult-onset Niemann-Pick disease type C patients treated with miglustat."
03/01/2015 - "Approximately half (n = 202) had GD1, and half had other diseases (mainly Niemann-Pick disease type C (NP-C), for which miglustat was approved in Europe in 2009). "
01/01/2015 - "Twenty-five patients with Niemann Pick disease type C (age range: 7 months to 44 years) were enrolled in an Italian independent multicenter trial and treated with miglustat for periods from 48 to 96 months. "
|3.||Neurologic Manifestations (Neurological Manifestations)
11/01/2008 - "Miglustat does not appear to have significant benefits on the neurological manifestations of GD3. "
01/01/2014 - "In a randomized clinical trial, long-term extension studies and a retrospective observational cohort study, treatment with oral miglustat stabilized key neurological manifestations of NP-C (including horizontal saccadic eye movement peak velocity, ambulation, manipulation, language and swallowing) in paediatric and adult patients with the disease. "
07/01/2012 - "Miglustat was subsequently approved in Europe to treat progressive neurologic manifestations in both children and adults in early 2009, based on clinical study data. "
12/01/2013 - "Therapeutic interventions are few but one that is of interest is miglustat, which has been approved for specific treatment of the neurological manifestations. "
09/10/2012 - "Miglustat is the first effective therapy for the neurological manifestations of NP-C patients, as it can slow down the progression of the disease. "
01/01/2014 - "This study aimed to probe the molecular basis for the anti-inflammatory activity of miglustat by examining specifically the role of GBA2 following the infection of CF bronchial epithelial cells by P. "
04/01/1993 - "Mechanism of action of N-butyl deoxynojirimycin in inhibiting HIV-1 infection and activity in combination with nucleoside analogs."
01/01/2014 - "Miglustat, a well-characterized iminosugar-based inhibitor of β-glucosidase 2 (GBA2), has shown promise in CF treatment because it reduces the inflammatory response to infection by P. "
02/01/1994 - "The safety and efficacy of combination N-butyl-deoxynojirimycin (SC-48334) and zidovudine in patients with HIV-1 infection and 200-500 CD4 cells/mm3."
01/01/2015 - "Thus, I propose a hypothetical treatment protocol to treat Ebola virus infection with a cumulative use of both Miglustat and Toremifene to inhibit the virus effectively and synergistically. "
|5.||Deglutition Disorders (Dysphagia)
03/01/2011 - "This observational study reports on the long-term use of miglustat in four pediatric patients with NPC and shows the efficacy of the treatment to improve or prevent dysphagia, and persistence after 3 years of treatment or more. "
01/01/2013 - "Swallowing function was stabilised during miglustat therapy, with no significant increase in Han dysphagia scale or aspiration-penetration index scores among four evaluable patients (p > 0.05). "
01/01/2013 - "Overall, 3/5 patients displayed progressive dysphagia before starting miglustat, and 4/5 showed marked cognitive and/or motor impairment. "
01/01/2012 - "Dysphagia as a risk factor for mortality in Niemann-Pick disease type C: systematic literature review and evidence from studies with miglustat."
03/01/2011 - "The videofluoroscopic swallowing study shows a sustained improvement of dysphagia in children with Niemann-Pick disease type C after therapy with miglustat."
|5.||Dietary Sucrose (Table Sugar)
|2.||Enzyme Replacement Therapy