Rett syndrome

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.
Rett syndrome Microphone

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 | 7 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
Illustration of a series of pills in petri dishes laid out to look like a descending line graph.
Spectrum Microphone

Biotech downturn hurts companies targeting autism-linked conditions

After a year of intense growth, funding for biotech is in decline. The result is layoffs and program cuts — and maybe some innovation.

By Angie Voyles Askham
23 February 2023 | 7 min read

Explore more from The Transmitter

Portrait of Kaspar Podgorski standing in his lab wearing a helmet with a climbing rope over his shoulder.

Climbing to new heights: Q&A with Kaspar Podgorski

The optical physiologist tracks neural computations inside the lab and scales sheer rock faces outside—even after a life-changing fall.

By Elissa Welle
21 June 2024 | 8 min read
Research illustration groups genes by their effects on brain cell types.

Giant analysis reveals how autism-linked genes affect brain cell types

Genes that predispose people to autism account for a large portion of the neuronal and glial cell changes seen in those with the condition.

By Charles Q. Choi
20 June 2024 | 5 min read

Widely used calcium imaging protocol can lead to spurious results, new paper cautions

The technique, which measures calcium currents as a proxy for neuronal firing, sometimes reports unusual and potentially misleading waves of activity in the hippocampus.

By Angie Voyles Askham
19 June 2024 | 0 min watch