FDA Grants Orphan Drug Designation to AND017 for the Treatment of Sickle Cell Disease
October 28th 2024Results demonstrating safety and efficacy in those with anemia in non-dialysis dependent chronic kidney disease and anemia in dialysis-dependent chronic kidney disease were presented at the 2024 American Society of Nephrology Kidney Week.
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Lodoco: A New Indication for an Old Drug
Historically, colchicine has been used primarily in the management of gout; however, in 2023, the FDA approved Lodoco, a newly marketed colchicine product, for the management of cardiovascular disease.
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FDA Grants Orphan Drug Designation to MDL-101 for Congenital Muscular Dystrophy Type 1a
Published: October 25th 2024 | Updated: October 25th 2024Congenital muscular dystrophy type 1a is a severe, early-onset condition that currently has no treatment, but MDL-101 may serve as a 1-time, durable treatment option.
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Sara Rogers, PharmD, discusses the formation and goals of the Standardizing Laboratory Practices in Pharmacogenomics (STRIPE) initiative, which seeks to standardize pharmacogenomic practices and improve clinical decision-making by establishing consensus across all stakeholders.
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Ryan Nelson, PharmD, discusses the STRIPE Annual Meeting and Consensus Workshop’s focus on unifying pharmacogenetic guidelines across major organizations, such as the FDA, European Medicines Agency, Clinical Pharmacogenetics Implementation Consortium, and National Comprehensive Cancer Network.
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