“For example, people who have epilepsy caused by mutations in the gene SCN1A should not be prescribed sodium channel blockers, as this can make their epilepsy worse. Another example is ...

in the SCN1A gene. Zorevunersen is being developed as potentially BEDFORD, Mass., December 02, 2024--Stoke Therapeutics, Inc. (Nasdaq: STOK), a biotechnology company dedicated to restoring protein ...

The TP53 gene is a gene that is mutated in many cancers. It is the most common gene mutation found in cancer cells. A tumor-suppressor gene, TP53 codes for a protein that inhibits the development and ...

Gene expression is the generation of a functional gene product from the information encoded by a gene, through the processes of transcription and translation. Gene products are often proteins ...

Jan. 28, 2025 — A team of researchers has developed a novel method to enhance the precision of cancer treatment using gold nanoparticles tagged with DNA ...

The table below is a review of notable updates that occurred in December 2024 for investigational products in development (not an inclusive list). Click on the status to view our full coverage.

An in vivo base editing approach targeting the PRNP gene led to a 52% increase in the lifespan of mouse models inoculated with the most common sporadic and genetic types of human pathogenic prion ...

Jan. 22, 2025 — Doctors could soon reduce epilepsy misdiagnoses by up to 70% using a new tool that turns routine electroencephalogram, or EEG, tests that appear normal into highly accurate ...

3 4 However, when no positive result is acquired from the CMA test, most patients usually resort to further gene sequencing to detect small genetic variants such as single nucleotide variations (SNVs) ...

Notable updates occurred in December 2024 for investigational products in development. The table below is a review of notable updates that occurred in December 2024 for investigational products in ...

Value-Creating Catalysts Anticipated Every Quarter in 2025: Q1 – FDA Decision on File Acceptance of Pitolisant sNDA for Idiopathic Hypersomnia; Potential Approval in 2025 Q2 – Orexin/BP1.15205 ...

The paper investigates a potential cause of a type of severe epilepsy that develops in early life because of a defect in a gene called KCNQ2. The significance is fundamental because it substantially ...