Researchers have discovered a potential “window” whereby babies born with inherited diseases may be treated with gene therapy sent directly into their circulatory systems. The window consists of circulating stem…
In 2014 the U.S. experienced a nationwide outbreak of EV-D68, raising concerns about children’s respiratory health. Acute flaccid myelitis disease (AFM) also spiked. EV-D68 is defined as a group of…
This week, the FDA approved the first blood-based diagnostic test for Alzheimer's disease (AD). The test, known as "Lumipulse G pTau 217/β-Amyloid 1-42 Plasma Ratio," is designed to detect amyloid…
Current research indicates that children and adolescents who were unvaccinated during the pandemic were much more likely to develop Long COVID. Researchers led by Perelman Medical School at the University of…
Patient Worthy is grateful to present this story written by Bud Hager by way of the Courageous Parents Network. CPN is a nonprofit organization that has oriented, equipped and empowered…
This article is written by Enrico Orzes and originally published on Osservatorio Malattie Rare (OMaR). It has been translated from Italian. Patient Worthy is grateful to the team at OMaR…
Novo Nordisk plans to seek approval in the US and the EU this year for their drug Mim8 for use on pediatric patients with Hemophilia A after promising results from…
Mesoblast Biotechnology Company has received FDA approval for its Ryoncil therapy as the first of its kind to be approved in the United States. The therapy has been developed to…
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