Cipaglucosidase alfa-atga: Unveiling new horizons in Pompe disease therapy

Cipaglucosidase alfa-atga: Unveiling new horizons in Pompe disease therapy

Pompe disease is a lysosomal storage disease characterized by impaired glycogen breakdown due to an acid α-glucosidase (GAA) enzyme deficiency. Without therapy, children with the severe infantile form do not survive past their first year of life. POMBILITI which is intended to treat late-onset Pompe...

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Bibliographic Details
Main Authors: Arshdeep Singh, Rabin Debnath, Aniket Saini, Kushal Seni, Anjali Sharma, Deepak Singh Bisht, Viney Chawla, Pooja A Chawla
Format: Article
Language:English
Published: Elsevier 2024-06-01
Series:Health Sciences Review
Subjects:
Pompe disease
Cipaglucosidase alfa-atga
Pombiliti
Clinical trials
Available treatment
Online Access:http://www.sciencedirect.com/science/article/pii/S2772632024000138

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http://www.sciencedirect.com/science/article/pii/S2772632024000138

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