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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.

April 2023
Long-tailed macaque in the canopy of a fruiting strangler fig tree, surrounded by ripening figs. Gunung Palung National Park, Borneo.

RNA therapy restores gene function in monkeys modeling Angelman syndrome

by  /  7 April 2023

The result raises hopes for an ongoing clinical trial in people — and offers fresh insight into the biology of imprinting and the UBE3A antisense transcript.

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Research image of radial glial cells in a human brain organoid.

Immune molecule alters cellular makeup of human brain organoids

by  /  5 April 2023

The changes may help explain the link between maternal infection and autism, though more research is needed.

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March 2023
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.

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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.

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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.

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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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