Rett Syndrome News

May 29, 2020May 29, 2020

Blocking P2X7 Receptors May Be Therapeutic Target for Rett

Rett Syndrome News

Blocking the activity of pro-inflammatory P2X7 receptors — a class of receptors found in immune cells and in glial cells (cells that support and protect neurons) — can reduce brain ... Read more

May 22, 2020May 26, 2020

Scientists Who Discovered Gene Link with Rett Syndrome Awarded 2020 Brain Prize

COVID-19 News, Rett Syndrome News

The scientists who discovered the MECP2 gene and its role in Rett syndrome have been awarded the 2020 Brain Prize, the world’s largest neuroscience prize granted by the Lundbeck Foundation. Sir ... Read more

May 15, 2020May 15, 2020

Taysha Raises $30M, Partners with UT Southwestern to Develop Gene Therapies for Rett, Other Disorders

Rett Syndrome News

Taysha Gene Therapies has raised $30 million and established a partnership with UT Southwestern Medical Center to expand its gene therapy program for neurological disorders caused by mutations in a ... Read more

May 8, 2020May 8, 2020

Newron Terminates Sarizotan Development Due to STARS Trial Failure

Rett Syndrome News

Treatment with sarizotan failed to reduce apnea episodes (breathing stops) while awake compared to a placebo, failing to achieve the primary goal of the STARS Phase 2/3 clinical trial in ... Read more

April 24, 2020April 24, 2020

Therapy Using ‘Unstable’ Gene Seen to Help Mice in Rett Model

Rett Syndrome News

A form of gene therapy based on providing an unstable version of the MECP2 gene extended survival and improved motor function in a mouse model of Rett syndrome, a study ... Read more

April 17, 2020April 17, 2020

Altered Nerve Cell Communication Tied to Motor Difficulties of Rett

Rett Syndrome News

Problems with nerve cell communication and adaptation in the brain’s motor cortex is associated with the severity of motor difficulties in people with Rett syndrome, a study shows. The research ... Read more

April 10, 2020April 10, 2020

Differences in Metabolism May Aid Rett Syndrome Characterization and Treatment

Rett Syndrome News

A comprehensive metabolic analysis uncovered significantly different levels of 66 molecules in people with Rett syndrome compared to their non-affected siblings, potentially providing a foundation to help better understand and ... Read more

April 6, 2020May 10, 2020

NORD’s Advice on COVID-19: You’re Stronger Than You Think

COVID-19 News, Rett Syndrome News

First, the bad news: If you’re one of the 30 million or so Americans with a rare disease, you probably have lower immunity to the novel coronavirus than most people. ... Read more

April 3, 2020May 8, 2020

Rett Syndrome Trial of Oral Anavex 2-73 to Continue with at-Home Visits

COVID-19 News, Rett Syndrome News

Anavex Life Sciences announced plans to continue to screen and enroll patients into its active trials for Rett syndrome and Alzheimer’s disease, in accordance to institutional rules and government guidance related to ... Read more

April 2, 2020May 2, 2020

NORD’s Advice on COVID-19: You’re Stronger Than You Think

Rett Syndrome News

First, the bad news: If you’re one of the 30 million or so Americans with a rare disease, you probably have lower immunity to the novel coronavirus than most people. ... Read more

March 27, 2020March 28, 2020

Researchers Shed Light on Link Between IGF-1 and Potential Benefits in Rett Syndrome

Rett Syndrome News

Treatment with insulin-like growth factor 1 (IGF-1) eases symptoms of Rett syndrome by suppressing the activity of FXYD1 — a molecule found in higher-than-normal levels in Rett patients — according ... Read more

March 20, 2020March 21, 2020

Amicus Opens Gene Therapy Center Focused on Rett Syndrome, Other Rare Diseases

Rett Syndrome News

Amicus Therapeutics has opened its Global Research and Gene Therapy Center of Excellence that will seek to advance research programs in Rett Syndrome and several other rare diseases. Besides Rett, ... Read more

March 17, 2020March 21, 2020

Newron Pharma Receives FDA Communication on STARS Trial; Results Expected Soon

Rett Syndrome News

Newron Pharmaceuticals has received the final minutes from a meeting with the U.S. Food and Drug Administration (FDA) regarding the pivotal STARS Phase 2/3 clinical trial investigating sarizotan as a ... Read more

March 12, 2020March 22, 2020

Pediatric Rare Disease Therapies Increase, But Most Repurposed, Study Finds

Rett Syndrome News

The number of treatments for children with rare diseases has grown over the past decade, according to a new study. However, despite the increase, nearly 7,000 rare diseases are still lacking ... Read more

March 10, 2020March 21, 2020

MECP2 Mutation Type Predicts Rett Syndrome Bone Disease Severity, Study Finds

Rett Syndrome News

The type of mutation in the MECP2 gene in people with Rett syndrome is a strong predictor of bone disease severity, a study suggests. Patients with more severe mutations in ... Read more