Hearing Loss Affects Nearly Two-Thirds of Iranian Children with Mucopolysaccharidosis
A cross-sectional study of 70 Iranian children with mucopolysaccharidosis found hearing loss in 64.29% of patients, with no significant association ...
Tag: enzyme replacement therapy
Enzyme Replacement Therapy Proves Safe and Stable in Chinese Patients with Fabry Disease
A 52-week phase 3 trial across six Chinese centers found agalsidase alfa was generally well tolerated, kept kidney and cardiac ...
Experts Project Lower Lifelong Organ Damage Risk With Recombinant ADAMTS13 in Rare Blood Clotting Disorder
A structured expert elicitation study projects substantially lower long-term organ damage risks for congenital thrombotic thrombocytopenic purpura patients receiving recombinant ...
Asfotase Alfa: 10 Years of Hypophosphatasia Insights
In a significant development for the treatment of hypophosphatasia (HPP), groundbreaking research has emerged detailing the effectiveness of Asfotase Alfa—a ...
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Mothers who receive childcare support from maternal grandparents show more parental warmth, finds NTU Singapore study
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University of Seville Breaks 120-Year-Old Mystery, Revises a Key Einstein Concept
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Bee body mass, pathogens and local climate influence heat tolerance
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Researchers record first-ever images and data of a shark experiencing a boat strike
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Groundbreaking Clinical Trial Reveals Lubiprostone Enhances Kidney Function
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