Phase 1 clinical trial of CMP-CPS-001 in Urea Cycle Disorders (UCDs) ongoing, with dosing completed in two of four multiple ascending dose (MAD) cohorts; safety, pharmacokinetic, and pharmacodynamic ...
AHA expresses support the introduction of the Conrad State 30 and Physician Access Reauthorization Act (S. 709).
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Dr. Julia Dallman, Associate Professor of Biology at the University of Miami, alongside her research team, received a $65,000 grant from the SynGAP Research Fund (SRF). This funding supports their ...
The SynGAP Research Fund (SRF) dba Cure SYNGAP1, a 501(c)(3) nonprofit organization, has awarded a $65,000 grant to Dr. Julia ...
The development could pave the way for therapies for rare disorders including some forms of cancer. It also holds potential for therapies to treat immune system and neurodegenerative disorders, ...
Johns Hopkins Medicine laboratory scientists say they have developed a potential new way to treat a variety of rare genetic diseases marked by too low levels of specific cellular proteins. To boost ...
Such disorders, although rare, include some forms of cancer, and immune system and neurodegenerative disorders, including SYNGAP deficiency, which results in learning disabilities and autism ...
Through overexpression of miR-101b, we showed that miR-101b is able to regulate the expression of SynGAP1 in an hippocampal cell line. Moreover and consistent with a decrease of miR-101b, Wnt-5a ...
Synapses play a pivotal role in forming neural circuits, with critical implications for brain functions such as learning, memory, and emotions. Several advances in synaptic research have demonstrated ...