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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
NORD and Rosenau Family Research Foundation Launch Rare Research Launchpad to Introduce Students to Careers in Rare Disease Research
21+ hour, 35+ min ago (638+ words) Newswise NORD and Rosenau Family Research Foundation Launch Rare Research Launchpad to Introduce Students to Careers in Rare Disease Research Free, online learning series and career exploration toolkit introduce aspiring scientists to rare disease research through real-world case studies Newswise…...
Native Americans Face High Rates of Preventable Alzheimer???s Risk Factors, Study Finds
18+ hour, 4+ min ago (269+ words) A new study found that Native people experience high rates of four modifiable Alzheimer’s risk factors — limited access to education, hypertension, social isolation and depression — highlighting opportunities for preventive care and public health interventions to reduce dementia risk. Native people…...
Encoded raises $275M to advance Dravet syndrome gene therapy
21+ hour, 20+ min ago (397+ words) Funds will support expansion study of ETX101, manufacturing upgrades Written by Patricia Inácio, PhD | Sept. 15, 2026 Encoded Therapeutics has raised $275 million in Series F financing to support the clinical development of its experimental gene therapy ETX101 for infants and young children with Dravet…...
UNC Lineberger Aids Next-Gen Cancer Model Creation
21+ hour, 57+ 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
1+ day, 3+ hour 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…...
Amprion Launches SAAmplify®-αSYN Plus: The First & Only Commercially Available CSF Test to Detect Alpha-Synuclein Profile for Multiple System Atrophy
19+ hour, 3+ min ago (344+ words) Amprion to unveil test and new research across six presentations at 2026 International Congress of Parkinson’s Disease and Movement Disorders in Seoul SAN DIEGO--(BUSINESS WIRE)--A new innovation in the detection of MSA pathology is here. Amprion, a global leader…...
ZVRA: Rapid U.S. growth, global expansion, and a strong pipeline drive rare disease innovation
1+ day, 14+ hour 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, 21+ 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
1+ day, 18+ hour 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, 21+ hour 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......