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BioWorld - Wednesday, April 29, 2026
Home » Keywords » fragile X syndrome

Items Tagged with 'fragile X syndrome'

ARTICLES

Neurology/psychiatric

Connecta Therapeutics discloses 3-phenoxy-3-phenylpropanamine derivatives

Feb. 3, 2026
Connecta Therapeutics SL has discovered 3-phenoxy-3-phenylpropanamine derivatives described as potentially useful for the treatment of fragile X and Rett syndrome.
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Neurology/psychiatric

California scientists report patent for new tetracycline derivatives

Sep. 10, 2025
Researchers from Cyclone Therapeutics Inc. and the Scripps Research Institute have disclosed tetracycline derivatives reported to be useful for the treatment of fragile X syndrome, rheumatoid arthritis and Alzheimer’s disease.
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Digital handshake

Servier in $450M deal for Medicxi-funded phase II fragile X program

Sep. 8, 2025
By Nuala Moran
No Comments
Medicxi Ventures’ asset-focused investment model has scored another hit, in a $450 million deal with Servier SA, for KER-0193, an oral small molecule that is ready for phase II development in the treatment of the inherited autism spectrum disorder, fragile X.
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Genetic/congenital

TRPC1 mediates pathological plasticity in fragile X syndrome mice

July 18, 2025
No Comments

Fragile X syndrome (FXS), the most common inherited cause of intellectual disability and autism, is caused by silencing of the Fmr1 gene, leading to a lack of the FMRP protein, which regulates protein synthesis in neurons. One key pathway affected by FMRP loss is the metabotropic glutamate receptor 5 (mGluR5) signaling pathway, where activation of mGluR5 leads to excessive translation of several proteins involved in synaptic plasticity. 


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Illustration of human brain and dna
Neurology/psychiatric

Quralis signs agreements to further fragile X syndrome research

May 16, 2025
No Comments
Quralis Corp. has entered into a number of agreements with the aim of advancing the treatment of fragile X syndrome, a genetic condition caused by a mutation of a single gene – fragile X messenger ribonucleoprotein 1 (FMR1) – on the X chromosome.
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Neurology/psychiatric

New preclinical data on NKCC1 inhibitor IAMA-6 in neurological disorder models

April 3, 2025
Growing evidence exists on regulation of the chloride importer solute carrier family 12 member 2 (SLC12A2), also known as NKCC1, as a therapeutic approach to treat neurological disorders. Altered expression of NKCC1 leads to impaired intracellular chloride levels in neurons and imbalance in the excitatory-inhibitory axis in the brain.
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Dendritic cells
Neurology/psychiatric

GluN2B signaling regulates dendritic spine plasticity and reverses FXS phenotypes

March 7, 2025
Fragile X syndrome (FXS) is the most common inherited form of intellectual disability and a leading monogenic cause of autism, yet effective treatments remain elusive. Previous work showed that N-methyl-D-aspartate receptors (NMDARs) play a prominent pathophysiological role in FXS and other neurodevelopmental disorders.
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AI-generated image of astrocytes
Neurology/psychiatric

Astrocyte Kir4.1 channels altered in fragile X syndrome

May 21, 2024
Correcting the deficiency of potassium intake in astrocytes could improve the symptoms of fragile X syndrome (FXS).
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Brain and DNA

Neuren’s NNZ-2591 meets endpoints in phase II trial in Phelan-McDermid syndrome

Dec. 19, 2023
By Tamra Sami
Neuren Pharmaceuticals Ltd. plans to progress NNZ-2591 to phase III trials following positive top-line phase II results in children with Phelan-McDermid syndrome, a genetic neurodevelopmental disorder for which there are no approved treatments.
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Brain and DNA

Neuren’s NNZ-2591 meets endpoints in phase II trial in Phelan-McDermid syndrome

Dec. 18, 2023
By Tamra Sami
Neuren Pharmaceuticals Ltd. plans to progress NNZ-2591 to phase III trials following positive top-line phase II results in children with Phelan-McDermid syndrome, a genetic neurodevelopmental disorder for which there are no approved treatments.
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