https://www.sanofi.com/en/our-science/therapeutic-areas/rare-diseases-research-development
We’re advancing innovative treatments in rare diseases and blood disorders, leveraging RNAi, nanobody technology, and enzyme replacement therapies to address...
rare diseasesblood disordersampsanofi
https://www.bleeding.org/bleeding-disorders-a-z/types/other-factor-deficiencies
Discover ultra-rare bleeding disorders, including Factor I, II, V, VII, X, XI, XII, and XIII deficiencies. Learn about symptoms, treatments, and find support.
bleeding disordersrareoverviewsymptomstreatment
https://www.netcetera.com/stories/news/AI-diagnoses-rare-genetic-disorders.html
AI diagnoses rare genetic disorders: Phivea® for reliable and safe diagnosis of genetic disorders
genetic disordersairarereliablesafe
https://www.khaleejtimes.com/kt-network/fakeeh-university-hospital-sets-new-benchmarks-in-advanced-gene-therapy
Discover how Fakeeh University Hospital is setting new benchmarks in advanced gene therapy, offering cutting-edge treatments for rare genetic disorders
university hospitalgene therapyleadsadvancedrare
https://rarediseases.org/
Feb 9, 2026 - NORD is dedicated to supporting education, elevating care, advancing research, and driving policy for rare diseases
rare disordersnationalorganizationnord
https://www.azolifesciences.com/article/Advances-in-Gene-Silencing-and-Editing-for-Rare-Disorders.aspx
Significant progress in Huntington's disease treatment: AMT-130 therapy achieves 75% slowing of disease progression using innovative gene silencing...
rare disordersadvancesgeneediting
https://www.bleeding.org/educational-programs/education/rare-bleeding-disorders
Find valuable resources & support for families living with rare bleeding disorders at NBFF. Access educational events, webinars, publications, workshops...
educational programsbleeding disorderspeoplerare
https://www.ppd.com/therapeutic-expertise/neuroscience-clinical-research/rare-neurological-disorders/
Aug 15, 2025 - Experts at rare neurological disease clinical trial execution which require dedicated teams committed to helping patients in need of treatment options.
neurological disordersclinical trialrareresearchppd