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Rare Diseases & Genetics
Orphan conditions, registries, gene testing, and family support networks.
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Latest in Rare Diseases & Genetics
UNC Lineberger Aids Next-Gen Cancer Model Creation
1+ hour, 38+ min ago (118+ words) Katherine A. Hoadley, PhD, a cancer genomics researcher at the UNC Lineberger Comprehensive Cancer Center, helped analyze data and plan and oversee a large international study that created one of the largest collections of cancer models grown from patient tumors. Published…...
Kala Bio and Virotek partner on US ophthalmic genetic testing
7+ hour, 6+ min ago (440+ words) Kala Bio, Inc. and Virotek, Inc. have finalized a definitive agreement in which Kala will be appointed the exclusive U.S. distributor and reseller of Virotek’s ophthalmic genetic testing and screening program. The companies first made their announcement last week by entering…...
NORD and Rosenau Family Research Foundation Launch Rare Research Launchpad to Introduce Students to Careers in Rare Disease Research
1+ hour, 22+ min ago (76+ words) Sep 15, 2026, 12:34 ET Free, online learning series and career exploration toolkit introduce aspiring scientists to rare disease research through real-world case studies The Rare Research Launchpad includes: Rare Research Launchpad is designed as a scalable model that can expand over time…...
ZVRA: Rapid U.S. growth, global expansion, and a strong pipeline drive rare disease innovation
18+ hour, 20+ min ago (74+ words) TradingView Focused on redefining rare disease care, the company has rapidly grown its U.S. market for Niemann-Pick type C, expanded globally, and advanced a promising pipeline. Strong financials and robust patient support underpin its strategy, with key regulatory and clinical milestones…...
RISE: Genetic Alliance Unveils a New Name and Identity for Its Global Rare Disease Genomics Network
1+ day, 1+ hour ago (131+ words) EIN Presswire There were 1,930 press releases posted in the last 24 hours and 487,780 in the last 365 days. RISE: Genetic Alliance Unveils a New Name and Identity for Its Global Rare Disease Genomics Network RISE — Rare Insights, Solutions, Empowerment — reflects a network…...
Scholar Rock Drug Wins First-in-Class Approval in Rare Neuromuscular Disease
22+ hour, 29+ min ago (308+ words) Scholar Rock’s Isembyld isn’t the only FDA-approved therapy for spinal muscular atrophy, but it is the first one that directly targets muscle tissue. Isembyld is the first commercial product for Scholar Rock, which is also evaluating the antibody in other…...
New multi-ancestry genetic score improves risk prediction for a common inherited heart muscle disease
2+ day, 56+ min ago (601+ words) A multi-ancestry polygenic risk score improved HCM risk prediction in a diverse U.S. population of more than 258,000 people. People in the highest genetic risk group had a 2.11-fold higher risk of HCM......
Beyond the First-Line for ESR1-Mutated Disease
2+ day, 21+ hour ago (273+ words) An expert panel moderated by Sara Nunnery, MD, of Tennessee Oncology, convened to discuss how they choose therapy beyond the first-line setting for patients with ESR1-mutated disease. Dr. Nunnery was joined by Nan Chen, MD, of the University of Chicago;…...
Common Lupus Gene Variant Boosts Antiviral Defenses at the Cost of
2+ day, 14+ hour ago (778+ words) Why do genetic variants that raise the risk of autoimmune disease remain so common in the human population? Evolutionary logic would seem to argue that harmful versions of genes should slowly disappear, yet many of the inherited risk factors for…...
ROS1 Fusion Subtype Shapes Survival in Advanced Lung Cancer, Real-World
2+ day, 18+ hour ago (599+ words) A rare but pivotal genetic alteration in lung cancer is once again in the spotlight, and this time the story is told through the lens of everyday clinical practice rather than the polished conditions of a randomized trial....