miglustat (Zavesca)

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. "
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."
01/01/2003 - "In noncomparative trials in patients with type 1 Gaucher's disease, miglustat (50 or 100mg three times daily) for 6-12 months significantly reduced baseline liver and spleen volumes. "

02/27/2015 - "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."
12/01/2020 - "Association of Miglustat With Swallowing Outcomes in Niemann-Pick Disease, Type C1."
01/01/2020 - "Miglustat has been indicated for the treatment of Niemann-Pick disease type C (NP-C) since 2009. "
01/01/2020 - "Long-term survival outcomes of patients with Niemann-Pick disease type C receiving miglustat treatment: A large retrospective observational study."

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. "
01/01/2023 - "While miglustat can stabilize neurological manifestations in later onset forms of NP-C, its efficacy in the early-infantile neurological form has not been demonstrated. "
06/01/2018 - "Miglustat treatment might improve or stabilize neurological manifestations but there is still limited data on the long-term efficacy. "
01/01/2018 - "Miglustat is indicated for the treatment of progressive neurological manifestations in both adults and children. "

01/01/2023 - "There is currently no cure for NP-C, and the only disease modifying treatment, miglustat, slows disease progression but does not significantly attenuate neurological symptoms. "
01/01/2019 - "A significant reduction in annual disease progression in infantile patients, and a trend towards reduced disease progression in juvenile patients after ≥1 year of miglustat treatment, translated into higher age at last contact or death in these groups. "
01/01/2018 - "There is no cure for NP-C but there is a disease-modifying drug (miglustat) that slows disease progression but with associated side effects. "
05/01/2024 - "Notably, all patients showed rapid disease progression despite treatment with miglustat before IT-HPBCD treatment. "
12/01/2020 - "Miglustat, a drug used off-label in the United States for the treatment of NPC1, appears to stabilize neurologic disease progression. "

04/01/2024 - "Long-term safety and efficacy of cipaglucosidase alfa plus miglustat in individuals living with Pompe disease: an open-label phase I/II study (ATB200-02)."
05/01/2024 - "104-week efficacy and safety of cipaglucosidase alfa plus miglustat in adults with late-onset Pompe disease: a phase III open-label extension study (ATB200-07)."
08/23/2023 - "Improved Enzyme Replacement Therapy with Cipaglucosidase Alfa/Miglustat in Infantile Pompe Disease."
04/01/2024 - "Cipaglucosidase alfa plus miglustat (cipa + mig) is a novel, two-component therapy for Pompe disease. "
11/01/2023 - "SIGNIFICANCE STATEMENT: This work describes important new insights into the treatment of Pompe disease using currently approved enzyme replacement therapies (ERTs) coadministered with miglustat. "