Multi-pronged media campaign to raise awareness about the disease and its impact on patients, family members and caregivers.Interactive, stand-alone NETHERTON NOW website launched.Quoin’s QRX003 product is on track to obtain the first regulatory approval for Netherton Syndrome. ASHBURN, Va., Feb. 04, 2025 (GLOBE NEWSWIRE) -- Quoin Pharmaceuticals Ltd. (NASDAQ: QNRX) (the “Company” or “Quoin”), a clinical-stage specialty pharmaceutical company focused on rare and orphan diseases, has launched the “NETHERTON ...

SUNNYVALE, Calif., Jan. 31, 2025 (GLOBE NEWSWIRE) -- 23andMe Holding Co. (Nasdaq: ME), a leading human genetics company with a mission to help people access, understand, and benefit from the human genome, today released a new polygenic risk score (PRS) report* on the genetics of osteoporosis for 23andMe+ Premium members. The report informs customers if they are at a higher likelihood of developing osteoporosis based on a statistical model developed by 23andMe through its proprietary research database, along ...

CALGARY, Alberta, Dec. 12, 2024 (GLOBE NEWSWIRE) -- XORTX Therapeutics Inc. ("XORTX" or the “Company”) (NASDAQ: XRTX | TSXV: XRTX | Frankfurt: ANU), a late-stage clinical pharmaceutical company focused on developing innovative therapies to treat progressive kidney disease, is pleased to announce a presentation by Dr. Allen Davidoff at the Rare and Genetic Kidney Disease Summit, in Boston, Massachusetts at 10:30 am ET, Thursday December 12, 2024. The presentation entitled “Autosomal Dominant Polycystic Kidne ...

On Tuesday, Spruce Biosciences, Inc. SPRB revealed topline results from its CAHmelia-204 study of tildacerfont in adult CAH and its CAHptain-205 study of tildacerfont in adult and pediatric CAH.Congenital adrenal hyperplasia (CAH) is a genetic disease that affects the adrenal glands and prevents the production of hormones like cortisol, aldosterone, and androgens.Also Read: Nano-Cap Spruce Biosciences Collaborates With HMNC Brain Health To Develop Treatment for Major Depressive Disorder“In the interim, the ...

TORONTO, Nov. 14, 2024 (GLOBE NEWSWIRE) -- The ALS Society of Canada (ALS Canada) is proud to announce Canada’s first National Genetic Counsellor dedicated to supporting people affected by amyotrophic lateral sclerosis (ALS), including people with a family history of the disease. Thanks to a $265,000 donation from TD Bank Group (TD), the two-year pilot project aims to provide access to crucial services delivered through virtual care, helping individuals understand their genetic risk and navigation of potent ...

Avidity expands its leading RNA delivery technology into precision cardiology with two new wholly-owned development candidates targeting rare genetic cardiomyopathies: AOC 1086 to treat PLN Cardiomyopathy and AOC 1072 to treat PRKAG2 Syndrome AOC 1072 and AOC 1086 preclinical data demonstrated robust siRNA delivery to the heart and targeted knockdown with potent reduction of approximately 80% in cardiac PLN mRNA and PRKAG2 mRNAAvidity introduces next-generation technology innovations demonstrating improved  ...

Dose-dependent increase in neuroprotective APOE2 expression in all participants with ongoing durability at 12 months Consistent reductions across CSF tau biomarkers and tau PET in majority of participants LX1001 well tolerated across all dose cohorts with no reports of amyloid-related imaging abnormalities (ARIA) Company to host webcast today at 7:00 AM ET NEW YORK, Oct. 30, 2024 (GLOBE NEWSWIRE) -- Lexeo Therapeutics, Inc. (Nasdaq: LXEO), a clinical stage genetic medicine company dedicated to pioneering tr ...

Myriad's MyRisk® test is the first hereditary cancer test integrated into Flatiron's OncoEMR® SALT LAKE CITY, Oct. 15, 2024 (GLOBE NEWSWIRE) -- Myriad Genetics, Inc. (NASDAQ: MYGN), a leader in genetic testing and precision medicine, today announced a collaboration with Flatiron Health, a leading health tech company dedicated to expanding the possibilities of point of care solutions in oncology. This collaboration allows physicians to order Myriad's MyRisk® Hereditary Cancer Test and view the results of the ...

NEW YORK, Oct. 15, 2024 (GLOBE NEWSWIRE) -- Tourmaline Bio, Inc. (Tourmaline) (NASDAQ: TRML), a late-stage clinical biotechnology company developing transformative medicines to dramatically improve the lives of patients with life-altering immune and inflammatory diseases, announced today that it will host a webinar, Understanding the Genetic Validation for IL-6 Inhibition in Cardiovascular Disease, featuring Dr. Dipender Gill, CEO and Founder of Sequoia Genetics. The webinar will be held on Friday, November ...

CALGARY, Alberta, Oct. 09, 2024 (GLOBE NEWSWIRE) -- XORTX Therapeutics Inc. ("XORTX" or the "Company") (NASDAQ: XRTX | TSXV: XRTX | Frankfurt: ANU), a late-stage clinical pharmaceutical company focused on developing innovative therapies to treat progressive kidney disease, is pleased to announce the Company has initiated a precision medicine program. On August 29, 2024, XORTX announced that independent peer-reviewed research reported that genetic factors are linked to the over-expression of xanthine oxidase ...