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• Northwestern University Study Suggests RNA Therapy Could Treat Rare Childhood Epilepsy Before Birth

  Published: May 1, 2026

  Section: Research

  Researchers have demonstrated that an experimental RNA-based treatment can calm abnormal brain activity associated with KCNT1-related epilepsy as early as 15 weeks gestation. By ta...

• Mount Sinai Researchers Identify Dysregulation of RNA Editing as a Primary Driver of Altered Fetal Brain Development in Down Syndrome

  Published: Mar 31, 2026

  Section: Research

  A multi-institutional study led by the Icahn School of Medicine at Mount Sinai has discovered that an extra copy of the ADARB1 gene on chromosome 21 leads to premature and excessiv...

• SynGAP Research Fund rebrands as CURE SYNGAP1 to accelerate therapeutic development for rare genetic disorders

  Published: Mar 21, 2026

  Section: Science & Tech

  The SynGAP Research Fund (SRF) has officially transitioned to the name CURE SYNGAP1 to better align its public identity with its mission of delivering disease-modifying treatments....

• Groundbreaking Discovery Of CD99L2 Gene Variants Solves Genetic Mystery Of X Linked Spastic Ataxia

  Published: Mar 17, 2026

  Section: Science & Tech

  A collaborative study between researchers in Bochum and Tübingen has identified the CD99L2 gene as a primary driver of rare movement disorders. By analyzing a cohort of nearly 3,00...