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Pharmacokinetics, safety and tolerability of miglustat in the treatment of pediatric patients with GM2 gangliosidosis.

Author: BanwellBrenda, ClarkeJoe T R, DingemanseJasper, MaegawaGustavo H B, MorganChristopher P, TifftCynthia J, YangSandra, van GiersbergenPaul L M

Clinical TrialA clinical trial is a planned study conducted on humans to evaluate the efficacy and safety of pharmaceuticals, medical devices, and treatment methods. It aims to explore the possibilities of new treatments and improve the health of patients.
Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Research Support, N.I.H., ExtramuralResearch funding provided by the NIH to external research institutions and researchers.
Research Support, Non-U.S. Gov'tResearch funding provided by government agencies outside the United States.

Overview

Miglustat, a substrate reduction therapy for GM2 gangliosidosis, is safe and well-tolerated in both infantile and juvenile patients. While mild to moderate diarrhea is a common side effect, it is manageable with dietary modification.
Paper Details　
Original Abstract of the Article :
GM2 gangliosidosis (GM2g) is an inherited neurodegenerative disorder caused by deficiency of lysosomal beta-hexosaminidase A, resulting in accumulation of GM2 ganglioside, principally in the brain. Substrate reduction therapy is currently under investigation as a treatment. The study investigated th...See full text at original site
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引用元：
https://doi.org/10.1016/j.ymgme.2009.04.013

データ提供：米国国立医学図書館（NLM）

Navigating the Desert of GM2 Gangliosidosis: Pharmacokinetics and Safety of Miglustat

The study delves into the complex world of GM2 gangliosidosis (GM2g), a rare and debilitating neurodegenerative disorder. It's like exploring a vast desert where GM2g poses a significant challenge to the body's health. The research focuses on miglustat, a medication used for treating GM2g, investigating its pharmacokinetics and safety in pediatric patients. It's like finding a potential oasis in the desert, a treatment that might offer relief and support for those affected by GM2g.

The researchers conducted a study involving pediatric patients with GM2g, administering miglustat in various doses and monitoring its pharmacokinetic properties and safety. It's like carefully navigating a desert landscape, adjusting the dosage of a precious resource (miglustat) to ensure its effectiveness and safety for young travelers.

The research found that miglustat was well-tolerated by pediatric patients with GM2g, with mild to moderate side effects. It's like finding a safe and reliable path through the desert, with some minor bumps and obstacles along the way, but ultimately a navigable route.

Miglustat: A Potential Oasis for GM2g Patients

The study suggests that miglustat can be a safe and effective treatment option for pediatric patients with GM2g. It's like discovering a hidden oasis in the vast desert of GM2g, offering a beacon of hope for those struggling with this debilitating disease. It's a significant finding that could improve the lives of individuals with GM2g, providing them with a valuable tool in their journey through the desert of illness.

Navigating the Desert of GM2g

GM2 gangliosidosis (GM2g) is a challenging disease, like a vast and unforgiving desert. This research offers a glimmer of hope, suggesting that miglustat could be a valuable tool in navigating the desert of GM2g, providing some relief and support for those affected. However, as always, it's important to consult with a healthcare professional for personalized guidance and to ensure the best possible treatment approach for your specific condition.

Dr.Camel's Conclusion

Miglustat emerges as a potential oasis in the challenging desert of GM2g, offering hope and support for pediatric patients. It's like finding a reliable camel to help navigate the difficult terrain of this debilitating disease.

Date :
  1. Date Completed 2009-09-11
  2. Date Revised 2014-11-20
Further Info :

Pubmed ID

19447653

DOI: Digital Object Identifier

10.1016/j.ymgme.2009.04.013

Related Literature

Article Analysis
・Side Effects of miglustat
SNS
PICO Info
in preparation
Languages

English

Positive IndicatorAn AI analysis index that serves as a benchmark for how positive the results of the study are. Note that it is a benchmark and requires careful interpretation and consideration of different perspectives.

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