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Guidance on the use of miglustat for treating patients with type 1 Gaucher disease.

Author: BarrangerJohn A, CharrowJoel, GrabowskiGregory A, MankinHenry J, MistryPramod, WeinrebNeal J

Comparative StudyResearch that compares multiple groups/interventions to determine differences/similarities in outcomes/characteristics.
ReviewAn article that critically examines existing research on a particular topic and summarizes the current state of knowledge in the field.
Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.

Overview

Type 1 Gaucher disease (GD) is a progressive lysosomal storage disorder. Intravenous enzyme replacement (ERT) with imiglucerase is the standard treatment for symptomatic patients. Miglustat, an oral treatment for patients with mild to moderate clinical manifestations, is an alternative treatment option for patients who are not candidates for ERT. Miglustat is associated with significant side effects and responses are slower and less robust than those observed with ERT.
Paper Details　
Original Abstract of the Article :
Type 1 Gaucher disease (GD) is a progressive lysosomal storage disorder due to an autosomal recessive deficiency of glucocerebrosidase. Clinical manifestations include anemia, thrombocytopenia, hepatosplenomegaly, and bone and pulmonary disease. Intravenous enzyme replacement (ERT) with imiglucerase...See full text at original site
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引用元：
https://doi.org/10.1002/ajh.20504

データ提供：米国国立医学図書館（NLM）

Type 1 Gaucher Disease: Seeking a Cure in the Desert of Genetic Disorders

The world of genetic disorders is a vast and unforgiving desert, with many diseases leaving patients struggling for relief. Type 1 Gaucher disease (GD) is one such condition, caused by a deficiency in the enzyme glucocerebrosidase. This research explores the treatment options for Type 1 GD, focusing on the use of miglustat, an oral medication, as an alternative to intravenous enzyme replacement therapy (ERT).

Navigating the Sands of Treatment Options: ERT vs. Miglustat

This research sheds light on the complex landscape of treatment options for Type 1 GD. While intravenous ERT is the standard treatment, miglustat offers an oral alternative for patients with mild to moderate symptoms. The authors analyze the benefits and risks of both approaches, providing valuable information for clinicians and patients navigating the desert of treatment options. The study highlights the importance of careful consideration when choosing the most appropriate treatment strategy for each individual.

Finding an Oasis of Hope: Treatment Options for Type 1 GD

This research offers hope for patients with Type 1 GD, providing a better understanding of the available treatment options. The authors provide a comprehensive overview of ERT and miglustat, highlighting their respective advantages and limitations. This information can empower patients to make informed decisions about their treatment and navigate the challenging journey of living with a genetic disorder, much like a camel navigating a vast desert with a clear roadmap.

Dr.Camel's Conclusion

The research on miglustat for Type 1 GD offers a glimmer of hope in the desert of genetic disorders. It highlights the importance of seeking a variety of treatment options and understanding their nuances to find the best fit for each patient. This research exemplifies the ongoing quest to improve the lives of those living with genetic diseases, a quest that continues to push the boundaries of medical knowledge and provide a beacon of hope in the darkness.

Date :
  1. Date Completed 2006-01-12
  2. Date Revised 2017-11-16
Further Info :

Pubmed ID

16247743

DOI: Digital Object Identifier

10.1002/ajh.20504

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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