Rett syndrome

Recent articles

Research image of toxoplasma gondii showing cysts it creates as well as the MECP2 protein that it can deliver to neurons.

Parasite-based tool delivers MECP2 and other proteins to neurons

A method that uses a common brain parasite could help replenish the proteins deficient in Rett syndrome and other conditions.

By Giorgia Guglielmi
12 September 2024 | 4 min read
A digital rendering of a strand of RNA

New RNA editor boasts increased versatility, safety

The “PRECISE” technique reprograms cells in a way that, unlike DNA editors, avoids potentially permanent off-target effects.

By Charles Q. Choi
11 April 2024 | 4 min read
Illustration of a sheet of red and white pills, with the red pills arranged in the form of a question mark.

New template streamlines sharing clinical trial results

This month’s Going on Trial newsletter explores a new tool for communicating with clinical trial participants and their families, among other drug development news.

By Calli McMurray
31 October 2023 | 7 min read
Illustration of a sheet of red and white pills, with the red pills arranged in the form of a question mark.

Trials test utility of EEG biomarkers for autism-related conditions

This month’s Going on Trial newsletter dives into an electroencephalography biomarker that could track the efficacy of treatments for dup15q and Angelman syndromes, among other drug development news.

By Calli McMurray
31 August 2023 | 7 min read
A hand reaches from above to add a pill to a stack that is resting against the x-axis of a graph.

How organ-on-a-chip models can help drug development

This month’s Going on Trial newsletter explores how organ-on-a-chip models could smooth the transition from preclinical to clinical trials, among other drug development news.

By Calli McMurray
31 July 2023 | 5 min read
Illustration of a sheet of red and white pills, with the red pills arranged in the form of a question mark.

Going on Trial: Gene therapy for Rett; return to arbaclofen

This month’s newsletter looks at the early safety data from the first gene therapy trial for Rett syndrome, among other drug development news.

By Calli McMurray
29 June 2023 | 6 min read
Illustration of a room with DNA sequence wallpaper and three doors leading to abstract new environments.

The future of autism therapies: A conversation with Lilia Iakoucheva and Derek Hong

If a therapy for autism’s core traits makes it to market, it will likely take one of three forms, the researchers say.

By Peter Hess
14 April 2023 | 5 min read
Drop of liquid medicine with air bubbles is dripping from a pipette on a light pink background.

FDA approval of trofinetide may spur further drug development for Rett

The drug, welcomed by patients, might be just the first of many.

By Shaena Montanari
16 March 2023 | 8 min listen
Illustration of a gloved hand reaching down to place a pill bottle on a pedestal.

Debut drug for Rett syndrome at edge of approval

The U.S. Food and Drug Administration plans to make an approval decision on the first-ever drug for girls and women with Rett syndrome by 12 March.

By Shaena Montanari
2 March 2023 | 9 min read
Illustration of a sheet of red and white pills, with the red pills arranged in the form of a question mark.

Going on Trial: Orphan drugs; CBD; bumetanide

This month’s newsletter takes a close look at the orphan drug program in the United States, several cannabis-based therapies and a secondary analysis of bumetanide, among other new developments in autism-related drug trials.

By Peter Hess
28 February 2023 | 7 min read

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A scientific fraud. An investigation. A lab in recovery.

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Illustration of hands sewing red and white threads in a DNA-like pattern into a blue-gray fabric.

Untangling biological threads from autism’s phenotypic patchwork reveals four core subtypes

People belonging to the same subtype share genetic variants, behaviors and often co-occurring diagnoses, according to a new preprint.

By Holly Barker
3 October 2024 | 5 min read
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Neural manifolds: Latest buzzword or pathway to understand the brain?

When you cut away the misconceptions, neural manifolds present a conceptually appropriate level at which systems neuroscientists can study the brain.

By Matthew Perich
2 October 2024 | 8 min read