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The National Organization for Rare Disorders (NORD) is applauding the Biden administration for announcing a rule to protect consumers from surprise medical billing, in a joint statement with 26 other U.S. patient organizations. The interim final rule will implement patient protections required by the No Surprises Act.
A first-generation university student has been awarded a six-year, $466,669 grant from the National Institutes of Health (NIH) to fund his doctoral and postdoctoral research into brain-derived neurotrophic factor (BDNF), a protein that plays a role in Rett syndrome. The Virginia Tech student, Raymundo Hernandez, called the NIH…
AllianceRx Walgreens Prime, a specialty and home delivery pharmacy, is partnering with TailorMed, a healthcare technology company, to help lower out-of-pocket prescription costs for specialty pharmacy patients. Medications attained through specialty pharmacies are those used to treat rare and chronic conditions in the U.S., and are often extremely costly.
Shape Therapeutics has raised $112 million in financing to support work into developing its portfolio of RNA technologies to potentially treat genetic diseases, including Rett syndrome. “We have witnessed the power of [messenger RNA] in the vaccine space and are now entering a new era where we…
Nominations are now open for the worldwide 2022 Black Pearl Awards from Eurordis-Rare Diseases Europe. The 12 award categories recognize individual advocates, policy makers, researchers, organizations, and companies who work to make a difference for the global rare disease community. The deadline for nominations is Sept.
The molecular mechanism by which the protein MeCP2 recognizes and binds to CA repeats — repetitive short sequences of DNA that are found throughout the genome — has been unraveled in a new study that sheds light on the causes of Rett syndrome. Researchers in France discovered that…
Treatment with a protein called CNF1 produced different results in two different mouse models of Rett syndrome, demonstrating a need to test therapeutic approaches across disease models, according to a study. Specifically, the CNF1 protein was able to rescue cognitive deficits, but not motor difficulties, in a mouse…
Using a virtual avatar to guide users during attention and memory tasks significantly improves the performance of people with Rett syndrome, according to a small study from Italy. The human-like interactive agent likely acts as a social motivator for these patients, who often focus on the face of parents…
The lack of a working MeCP2 protein — the most common cause of Rett syndrome — skews the immune system toward a proinflammatory state in the central nervous system (CNS, brain and spinal cord), a study in mice suggests. According to its scientists, this finding helps in understanding…
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