May 21, 2025
Lysosomal Storage Disorders, or LSDs, are rare genetic conditions that may not make headlines, but they affect thousands worldwide, often with life-changing consequences. LSDs happen when the body’s lysosomes, tiny cellular recycling centers, fail to break down waste materials due to missing or faulty enzymes. This...
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Sep 04, 2023
Fabry disease is a rare hereditary lysosomal storage disorder that is caused by mutation in the GLA gene located on the X chromosome. The defect leads to the deficiency of an enzyme called alpha-galactosidase A, responsible for the breakdown of globotriaosylceramide (Gb3). The accumulation of the fatty substance le...
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Mar 28, 2022
Fabry Disease is an inherited lysosomal storage disease caused by a nonfunctional or partially functional enzyme called alpha galactosidase A (α-Gal A). Decreased activity of α-Gal A in lysosomes results in the accumulation of enzyme substrates (Gb3 and lyso-Gb3) which cause cellular damage in tissues throughout th...
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Aug 17, 2018
Alnylam’s Onpattro Receives First-Ever US FDA Approval for Treatment of the Polyneuropathy of Hereditary Transthyretin-Mediated Amyloidosis in Adults US FDA approved Alnylam’s Onpattro (patisiran) lipid complex injection, a first-of-its-kind RNA interference (RNAi) therapeutic, for the treatment of the polyneurop...
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A New Era for Multiple Myeloma Treatment: Bispecific Antibodies Enter the Fray
Jun 10, 2025
LEQEMBI: A New Hope for Alzheimer’s Disease Patients
Feb 12, 2025
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