News

Unravel Biosciences has teamed up with Quality Chemical Laboratories (QCL) to manufacture materials for clinical trials of RVL001, an investigational therapy for Rett syndrome. RVL001 (vorinostat) will be tested in proof-of-concept trials in the U.S. and Colombia. Last year, the U.S. Food and Drug Administration (FDA)…

New research supports how the lack of Mecp2 protein impairs the function of healthy nerve cells in a mouse study of Rett syndrome. Specifically, the function of nerve cells was affected when the cells were grown alongside nerve-cell supporting astrocytes lacking the Mecp2 protein, mimicking the genetic defect that…

Neurogene is expanding its ongoing Phase 1/2 clinical trial testing gene therapy candidate NGN-401 in girls with Rett syndrome and making some changes to the trial’s structure to accelerate enrollment and dosing. Changes include adding more patients and removing dose staggering (one-at-a-time dosing) in the trial’s first low-dose…

Taysha Gene Therapies has received the needed approval to move forward with dose escalation in its Phase 1/2 trial testing TSHA-102, its gene therapy candidate for Rett syndrome, in adolescents and adults, the company announced. That green light from the study’s independent data monitoring committee (IDMC) — a group…

Sound processing — part of a complex system in the brain — is impaired in girls with Rett syndrome, and those impairments correlate with deficiencies in the children’s speech skills, a new study found. In their work with more than 40 children with Rett, a team of researchers in…

The use of low-dose extracorporeal shock wave therapy — a safe and noninvasive treatment known as ESWT — for three months was found to improve motor function in children with Rett syndrome. Reduced muscle stiffness, known as spasticity, also was seen among the children, but the benefits were not…

Taking a long time to fall asleep and having increased rapid eye movement (REM) sleep correlate with a higher burden of purposeless hand movements, called stereotypies, in girls with Rett syndrome, a new study shows. These findings may also suggest a “higher risk of insomnia in individuals with [Rett]…

An injection of Taysha Gene Therapies’ experimental gene therapy TSHA-102 into the spinal canal of young mice modeling Rett syndrome can improve weight, survival, breathing, and motor skills, a preclinical study suggests. The data support using the intrathecal route in ongoing Phase 1/2…

Among experienced physicians, neurologists are reportedly more comfortable than pediatricians in treating people with Rett syndrome, according to a survey conducted in the U.S. Neurologists also consider more tools than pediatricians do when diagnosing patients with Rett, data show. These findings highlight the need for “better education and support…

Up to nearly half of children and adults with Rett syndrome in the U.S. have digestive problems, resulting in added medical costs that average more than $4,400 per patient each year, a study finds. While gastrointestinal (GI) manifestations appear to represent a great burden…