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Spectrum: Autism Research News

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Treatments

Efforts to ease the symptoms of autism are beginning to ramp up, with promising candidates in various stages of testing.

Illustration of a single patient in a clinical trial sitting on a bed in a glitchy, imperfectly rendered room.

Lessons from n-of-1 trials: A conversation with Joseph Gleeson

by  /  30 March 2023
Some conditions are too rare for conventional drug trials, leading some scientists to test bespoke treatments in single participants. Gleeson discusses the merits — and limitations — of these tiny trials.
A hand reaches from above to add a pill to a stack that is resting against the x-axis of a graph.

Going on Trial: Trofinetide approval for Rett; n-of-1 ASO therapies; cord-blood deals

by  /  30 March 2023
This month’s issue of the Going on Trial newsletter examines personalized therapies for rare conditions, Acadia’s new drug for Rett syndrome and developments in a cord-blood program, among other autism-related drug trial news.
Computer-generated image of mountains against a black background.

Detecting a signal amid noise in autism early-intervention research

by , ,  /  28 March 2023
Studies of behavioral treatments for autism are complex and can easily be misunderstood. Here we provide some guidance.
March 2023
Research image of cultured neurons.

Cannabis compound rebalances signaling to quell seizures in mice

by  /  22 March 2023

Cannabidiol (CBD) blocks the action of a molecule that drives an overexcitability feedback loop in a rodent model of epilepsy.

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Adapted research image of immunofluorescence staining of primary cortical neurons showing expression of PTBP proteins and reduced level of the protein SYNGAP1.

‘Splice-switching’ strategy boosts SYNGAP1 expression

by  /  21 March 2023

The approach improves the function of SYNGAP1-deficient neurons in vitro, but whether it will work in people remains unclear.

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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

by  /  16 March 2023

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

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Illustration of a gloved hand reaching down to place a pill bottle on a pedestal.

Debut drug for Rett syndrome at edge of approval

by  /  2 March 2023

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.

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A bar graph with a hand extending from below to push the center bar higher.

Private equity harms autism service market

by  /  1 March 2023

Time is running out to expose this disreputable push for profit over care and change insurance, funding and training practices for the better.

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February 2023
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

by  /  28 February 2023

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.

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

Biotech downturn hurts companies targeting autism-linked conditions

by  /  23 February 2023

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

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A photograph of an infant's foot.

Dietary changes ease traits in rare autism-linked condition

by  /  21 February 2023

Early treatment with nutritional supplements and a high-protein diet forestalls some neurodevelopmental problems for children with BCKDK deficiency.

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Research image shows elevated expression of certain genes, shown in pink and green, along with expected gene expression, shown in blue.

Autism-linked MYT1L mutations prompt ‘identity crisis’ in budding brain cells

by  /  14 February 2023

Both human and mouse progenitor cells with the alterations struggle to become neurons and instead express genes that are typically active only in muscle or the heart.

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Picture of a caged mouse with an EEG implanted.

Adult Angelman mice get some benefit from boosting UBE3A gene expression

by  /  9 February 2023

The treatment eases the animals’ sleep troubles, suggesting it has clinically meaningful effects beyond what was thought to be a critical window in early life.

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