About one in 10 prescription drugs—mainly brand-name and expensive drugs and those for rare diseases—has a manufacturer-sponsored patient support program, which usually includes financial, nursing and educational supports.
Nov 27, 2023
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Much research has been conducted over many decades on diseases that are widespread in large parts of the population, such as cancer and heart disease. As a result, treatment methods have improved enormously thanks to long-term ...
Nov 24, 2023
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Vitamin B12 is a well-known micronutrient that has long been acknowledged for its essential role in maintaining nerve function, supporting red blood cell production, and facilitating DNA synthesis, all vital processes for ...
Nov 16, 2023
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The advent of whole genome sequencing technology has prompted an explosion in research into how genetics are associated with disease risk. But the vast majority of genetics research has been done on people of European ancestry, ...
Nov 15, 2023
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The U.S. Food and Drug Administration has approved Takeda Pharmaceuticals Adzynma, the first recombinant protein product indicated for prophylactic or on-demand enzyme replacement therapy (ERT) in adult and pediatric patients ...
Nov 14, 2023
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A new review article in Human Gene Therapy summarizes the significant milestones in the development of gene therapy medicinal products that have facilitated the treatment of a significant number of rare diseases. The article ...
Nov 13, 2023
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It's likely that few people had heard of frontotemporal dementia until earlier this year, when the family of actor Bruce Willis announced the 68-year-old had been diagnosed with the condition.
Nov 8, 2023
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Many hidden genetic variations can be detected with Chameleolyser, a new method developed in Nijmegen. The information is already yielding new patient diagnoses and may also lead to the discovery of as yet unknown disease ...
Nov 1, 2023
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In two publications in Nature Reviews Drug Discovery, Anneliene Jonker (TechMed Center) highlights the importance of health care for rare diseases, and the innovation needed in that. The personalized medicine program manager ...
Oct 25, 2023
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In a major step toward untangling the genetic complexities of rare diseases, KAUST researchers have unveiled an innovative AI-based tool that leverages varied symptom descriptions, along with evidence from the scientific ...
Oct 10, 2023
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