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The U.S. Food and Drug Administration (FDA) granted rare pediatric disease designation to trofinetide, a potential oral therapy for people with Rett syndrome. The designation provides priority review to investigational treatments with potential to provide clinically meaningful benefits in serious or life-threatening rare diseases mainly affecting individuals younger…
In recognition of Rare Disease Day Feb. 29, Bionews Services launched a social media campaign last month asking patients to describe what makes them rare. Running Feb. 7–29, the #WhatMakesMeRare campaign was aimed at uplifting people with rare diseases by encouraging them to share their stories and perspectives. The…
Restoring the levels of Wnt6, a signaling molecule involved in brain function, eased motor and behavioral difficulties in a mouse model of Rett syndrome, a study shows. These findings added to scientists’ knowledge of the underlying molecular mechanisms of Rett and may help develop new therapies for those…
Beginning on Feb. 29, Rare Disease Day, chapters from notable scientific books and clinical review articles covering rare disorders will be available free-of-charge from Elsevier. The offer runs through April 30, and aims to supports work by researchers and clinicians into a better understanding of and treatments for rare diseases, as well…
Starting a 501(c)(3) tax-exempt nonprofit isn’t easy, but the National Organization for Rare Disorders gave a few tips for those looking to begin the complex process in its Feb. 20 webinar. William Whitman…
An abundance of events are afoot around the world to mark Rare Disease Day 2020 on Feb. 29. The activities are focused on heightening awareness about rare diseases and the hundreds of millions of individuals they are thought to affect. Patients, caregivers, and advocates worldwide will sport denim ribbons…
Newron Pharmaceuticals is expanding the International Rett Syndrome Burden of Illness Survey to Europe and Australia in partnership with the worldwide Rett patient advocacy community, following strong participation from caregivers and medical professionals in the United States. Launched last November in the U.S., the survey is being extended…
High-quality speech therapy and more access to augmentative and alternative communication (AAC) tools are needed in schools for girls with Rett syndrome, according to a U.S. survey. The survey’s findings, “Special Education Supports and Services for Rett Syndrome: Parent Perceptions and Satisfaction,” were published in…
The U.S. Food and Drug Administration (FDA), a vast government bureaucracy, employs about 17,500 people and had a budget of $5.7 billion in 2019. Yet even with its enormous resources, the FDA these days relies more and more on patients to…
The gleaming new Dutch headquarters of the European Medicines Agency (EMA), fronting Domenico Scarlattilaan in Amsterdam’s suburban Zuidas business district, finally opened for business last month — just over two years after the European Union decided to relocate the EMA to the Netherlands in the wake of Brexit.
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