Positive Data Reported in a Multinational Clinical Trial Investigating New Treatment of Niemann-Pick disease Type C

A multinational clinical trial of for the treatment of Niemann-Pick disease Type C (NPC) demonstrated a statistically significant and clinically meaningful improvement in symptoms, functioning, and quality of life in both primary and topline secondary endpoints for both pediatric and adult patients with NPC.

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Composing New Proteins with Artificial Intelligence

Proteins are the building blocks of life and scientists have long studied how to improve them or design new ones. Traditionally, new proteins are created by mimicking existing proteins or manually editing their amino acids. This process is time-consuming, and it is difficult to predict the impact of changing an amino acid. In APL Bioengineering, researchers explore how to create new proteins by using machine learning to translate protein structures into musical scores, presenting an unusual way to translate physics concepts across domains.

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