Due to the unprecedented challenges caused by the ongoing COVID-19 pandemic, GW Pharmaceutics has stopped the ARCH Phase 3 clinical trial evaluating the oral cannabidiol (CBD) solution Epidiolex in children and adolescents with Rett syndrome. The decision was made after evaluating the progress of…
News
Leaders in the U.S. rare disease community came together recently for a webinar to present helpful information on how to start a nonprofit and patient registry. They shared about how their respective organizations came to be, as well as the benefits of creating patient registries and how they can help…
The transition into adulthood of women with Rett syndrome warrants a tailored program and a multidisciplinary team to provide the best clinical care, according to a 13-year Italian study of the complex symptoms experienced by these patients. Most adults with Rett deal with symptoms ranging from epilepsy and bone…
Problems with skilled learning tasks that involve motor function and making sense of sounds — known to be affected by Rett syndrome — were seen in rat model of the disease and consistent with these deficits in patients, a study reported. These findings could support work into…
Taysha Gene Therapies and Catalent have established a partnership to advance Taysha’s gene therapies for neurological disorders, including Rett syndrome. The partnership allows Taysha to use Catalent’s gene therapy facilities in Maryland to expand the company’s manufacturing capacity. The gene therapy for Rett, called TSHA-102, uses a modified,…
Of the people who know about Rett syndrome, many think it affects only females. But families of boys with this disorder know differently. Parents of children with an ultra-rare manifestation of this already rare and little-known disease find each other online and grow close in this…
The National Organization for Rare Disorders (NORD)’s RareLaunch training program will host two days of free virtual workshops in December, with the aim of empowering leaders to start non-profit organizations and research programs to help people with rare diseases. “The RareLaunch program is central to NORD’s mission and history — community…
As the Rett Syndrome Research Trust (RSRT) prepares to launch a global database of caregiver-provided information called The Rett Syndrome Global Registry, it is seeking families to participate in a pilot program to test its objectives. The goal in launching the registry is threefold: first, to provide a…
A photo of a bespectacled young boy, his red baseball cap slightly askew as he enjoys time outside, will be featured on the front cover of an upcoming calendar in the “Same But Different” contest to raise awareness about rare disorders. “A Lovely Day Out in Kew Gardens,”…
News
Antidepressant Mirtazapine Helps Preserve Motor Function in Rett Patients and Mice, Study Finds
Treatment with the antidepressant mirtazapine preserves motor and respiratory function, as well as social behavior, in women with Rett syndrome, a study shows. Improved motor function also was seen in a mouse model of the disorder. The study, “Protective role of mirtazapine in adult female…
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