In the study, the UCSF team demonstrated that restoring healthy levels of SCN2A expression through CRISPR activation, even in late development, could reverse several disease-relevant deficits present ...
CAMBRIDGE, Mass., April 30, 2021 (GLOBE NEWSWIRE) -- Praxis Precision Medicines, Inc. (NASDAQ: PRAX), a clinical-stage biopharmaceutical company translating genetic insights into the development of ...
A genetic change or variant in a gene called SCN2A is a known cause of infantile seizures, autism spectrum disorder and intellectual disability, as well as a wide range of other moderate-to-profound ...
The current study has been immediately converted from a double-blind, sham-controlled study to a single-arm, baseline-controlled study, enrolling 30 patients reduced from 40 patients. All patients ...
When brain development gets off to a bad start, the consequences are lifelong. One example is a condition called SCN2A haploinsufficiency, in which children are born with just one functioning copy of ...
A technology that activates healthy genes reduced seizures in a mouse model of a severe genetic neurological condition (1 Trusted Source Can CRISPR Fix a Childhood Brain Disorder? Go to source). When ...
Praxis Precision Medicines, Inc. (NASDAQ: PRAX), a clinical-stage biopharmaceutical company translating genetic insights into the development of therapies for ...
Having already claimed a win for the phase 2 study of its seizure drug, Praxis Precision Medicines is hoping fresh efficacy data will form the backbone of an approval application for the sodium ...
Could CRISPR Fix this Severe Childhood Brain Disorder? A technology that activates healthy genes reduced seizures in a mouse model of a severe genetic neurological condition. When brain development ...
Editor's Note: This story was originally published on October 20, 2016. After living for two years with SCN2A, a rare form of epilepsy, Charlotte Umezu died on November 29, 2016. Tracy Umezu notified ...
CAMBRIDGE, Mass. and SAN FRANCISCO, Sept. 17, 2025 /PRNewswire/ -- The laboratories of Dr. Kevin Bender and Dr. Nadav Ahituv at the University of California, San Francisco, today announced the ...
Detailed price information for Praxis Precision Medicines Inc (PRAX-Q) from The Globe and Mail including charting and trades.
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