Medicus Pharma To Participate in the 38th Annual Roth Conference

Medicus Pharma Ltd. (NASDAQ: MDCX) ("Medicus" or the "Company"), a biotech/life sciences company focused on advancing the clinical development programs of novel and potentially disruptive therapeutics ...

Atrium Therapeutics Launches with Approximately $270 Million to Advance Novel RNA Medicines for Rare Genetic Cardiomyopathies

Atrium Therapeutics, Inc. (Nasdaq: RNA) launched today as a newly independent, publicly traded company dedicated to delivering RNA therapeutics directly to the heart to transform care for people ...
Journal of Medical Genetics

Inherited variants in autosomal dominant disease genes are a significant cause of fetal structural anomalies

Background Monogenic disorders are a major cause of fetal structural anomalies. Most genetic diagnoses involve de novo, biallelic or X linked variants; however, inherited variants in autosomal ...
Morningstar

Stoke Therapeutics Announces First Patient Dosed in Phase 1 Study of STK-002, a Potential Disease-Modifying Medicine for the Treatment of Autosomal Dominant Optic Atrophy (ADOA)

Stoke Therapeutics Announces First Patient Dosed in Phase 1 Study of STK-002, a Potential Disease-Modifying Medicinefor the Treatment of Autosomal Dominant Optic Atrophy (ADOA) –Dose-escalating study ...
Metro

Ryan Searle defying hereditary eyesight issue at World Darts Championship

Ryan Searle is into the top 10 players in the world (Picture: Getty Images) Ryan Searle is into the World Darts Championship semi-finals despite an eyesight issue which impacts both him and his ...
BMJ

Genotype-aortic phenotype correlations in Marfan syndrome: a systematic review and meta-analysis of Fibrillin-1 variants

Introduction Marfan syndrome (MFS) is an autosomal dominant condition characterised by a wide array of pleiotropic manifestations that affect the cardiovascular, skeletal, ocular and pulmonary systems ...
Seeking Alpha

Calico Life Sciences Announces U.S. FDA Fast Track Designation for Investigational Treatment of Autosomal Dominant Polycystic Kidney Disease

SOUTH SAN FRANCISCO, Calif., Oct. 2, 2025 /PRNewswire/ -- Calico Life Sciences LLC (Calico), a biotechnology organization focused on aging and age-related diseases and founded by Alphabet Inc. and ...
MedPage Today

Video: Identifying Risk Of Kidney Function Decline In Autosomal Dominant Kidney Disease

Data from this Australian study showed a higher prevalence of intracranial aneurysms (ICAs) in patients with ADPKD compared with the general population. Many patients in the study had no known risk ...
mayo

A Study To Assess Adverse Events And Effectiveness Of IntraVenous Infusions Of ABBV-CLS-628 In Adult Participants With Autosomal Dominant Polycystic Kidney Disease (ADPKD)

Describes the nature of a clinical study. Types include: Observational study — observes people and measures outcomes without affecting results. Interventional study (clinical trial) — studies new ...
BioSpace

Novel Human Genetics Evidence Confirms Estimates of Genetic Prevalence, Underdiagnosis, and Potentially Greater Symptom Burden of Gain-of-Function CASR Variants Associated with ...

- By examining over 100 unique variants associated with the CASR gene causative of ADH1, the average frequency of gain-of-function CASR variants was ~3.7 per 100,000, closely aligned to previously ...
EurekAlert!

Hope in sight for autosomal dominant optic atrophy (ADOA)

Autosomal dominant optic atrophy (ADOA), the most common genetic optic neuropathy, is an insidious disease. It often presents slowly during childhood by way of blurry vision, trouble reading or ...