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Gene-Based Diagnosis 101: How to Successfully Navigate the Diagnostic Journey
As scientific innovation in genomic medicine continues to provide more hope for therapeutic options, there is a […]
Read moreA Case for Whole Genome Sequencing
The ability to diagnose rare genetic diseases through the use of genetic sequencing has improved to a […]
Read moreAdvances in Rare Immunological Diseases
Recent advances in rare immunological diseases have raised the exciting potential of cures for some disorders. Hear […]
Read moreDevelopmental Delays After Factor V Leiden Pregnancy
by Tiffany Burnette I was diagnosed with Factor V Leiden when I was pregnant with Sammy. While […]
Read morePatient Stories
A Family PKU Journey from Diagnosis as a Newborn to Living as an Adult
December 3rd is National PKU Awareness Day, and Global Genes asked the National PKU Alliance and a […]
Read moreWhat Those With Rare Disease, And Those Who Don’t Have In Common
by Megan Marjorie I remember during one of my many “solo” ICU stays I had this fantastic […]
Read moreMy Journey Becoming Stronger And Resilient Living With My Complex Medical Conditions
By Saida Mahoney Last month, I went to my neurologist and my neuromuscular doctor appointments. I have […]
Read moreWould You Like Some Water? Redefining Normal as a Phelan-McDermid Syndrome Parent
October 22nd is Phelan-McDermid Syndrome Awareness Day, and we asked a peer representative from the Phelan-McDermid Syndrome […]
Read moreSuper T’s Mast Cell Foundation continues Taylor’s vision to raise awareness about Mast Cell diseases
By Tammy Nearon RARE Mom & Advocate Taylor was the visionary behind Super T’s Mast Cell Foundation. […]
Read moreIt’s Time to Banish the Term “Abnormal” to Describe the External Physical Manifestations of Rare Diseases
by Kara Ayik Diverse, dissimilar, distinct, unlike, unalike, distinctive, uncommon, unique, another, not the same, characteristic, singular, […]
Read moreLatest News
SpringWorks Raises $275 Million in Upsized Public Offering
Rare Daily Staff One week after receiving U.S. Food and Drug Administration approval to market its desmoid […]
Read moreGlobal Genes’ Inaugural Open Science Data Challenge expands known phenotype for Rare Neurodevelopmental Disorders
The Inaugural Xcelerate RARE Open Science Data Challenge ran from May 31 – August 31, 2023, with […]
Read moreTop Tips for Newly Diagnosed with a RARE Disease
Getting a new diagnosis brings a flood of questions, uncertainty and unknowns. We asked our community who […]
Read moreDriving Awareness of Thyroid Eye Disease
Global Genes is excited to collaborate with Viridian Therapeutics and TED Community Organization through our Corporate Alliance […]
Read moreWhy Can’t People with RARE Diseases & Wheelchairs Fly Major Airlines?
Sign Open Letter to Improve Accessibility on Airlines As an organization founded to gather together advocates for […]
Read moreTop 10 Takeaways from the 2023 RARE Health Equity Forum
Whether you attended in person or watched the live stream, we hope that you now have ideas […]
Read moreIndustry News
Wave Life Sciences Prices $100 Million Public Offering of Ordinary Shares
Rare Daily Staff Wave Life Sciences, a clinical-stage RNA medicines company, priced an underwritten public offering of […]
Read moreRARE-X Launches Pompe Consortium
Rare Daily Staff RARE-X, Global Genes’ research program and data-sharing platform, launched The Pompe Consortium, a group […]
Read moreCIRM Issues $17.5 Million in Clinical Stage Research Funding
Rare Daily Staff The California Institute for Regenerative Medicine has awarded $17.5 million to fund clinical-stage research […]
Read moreHarmony Reports Positive Topline Data from Phase 2 Study in DM1
Rare Daily Staff Harmony Biosciences reported positive topline results from its phase 2 signal detection study evaluating […]
Read moreFDA Grants Fast Track Designation to Solid Biosciences for Duchenne Muscular Dystrophy Gene Therapy
Rare Daily Staff The U.S. Food and Drug Administration granted Fast Track designation to Solid Biosciences for […]
Read moreFDA Grants Priority Review for Ipsen and Genfit’s Treatment of Rare Cholestatic Liver Disease
Rare Daily Staff The U.S. Food and Drug Administration has accepted the New Drug Application and granted […]
Read moreAccelerating the Development of Genetic Medicines with AI
The advent of genetic medicines is enabling the development of therapies that can repair or replace a […]
Read moreFDA Approves Novartis Oral Therapy for PNH
Rare Daily Staff The U.S. Food and Drug Administration approved Novartis’ Fabhalta, the first oral monotherapy for […]
Read moreFDA Grants Breakthrough Therapy Designation for Neurocrine’s Crinecerfont in CAH
Rare Daily Staff The U.S. Food and Drug Administration granted Breakthrough Therapy designation to Neurocrine Biosciences for […]
Read moreGenetic Counseling
Top 10 Takeaways from the 2023 RARE Advocacy Summit
Did you LEVEL UP while attending or live streaming the 2023 RARE Advocacy Summit? We certainly did! […]
Read moreSeptember is Newborn Screening Awareness Month
Learn what newborn screenings are, why they are important, how regulations vary by states across the U.S., […]
Read moreBreakthroughs Begin with You: Cure SMA Celebrates Spinal Muscular Atrophy Awareness Month
August is Spinal Muscular Atrophy (SMA) Awareness Month, so Global Genes invited Cure SMA, a Global Advocacy […]
Read moreDiscovering Joy in Today: Nicki & Ganesh’s Story
by Nicki Bhandaru, RARE Mom My name is Nicki Bhandaru, and my son Ganesh has X-ALD. A […]
Read moreCEO Charlene Son Rigby Talks Drug Development and the Rare Research Roadmap
Charlene Son Rigby, CEO of Global Genes, talks about rare drug development, how patient advocates can contribute […]
Read moreMay is Ehlers-Danlos Awareness Month – Learn More About EDS
Lara Bloom, President and CEO of The Ehlers-Danlos Society, talks about why learning about Ehlers-Danlos Syndrome is […]
Read moreResearch Readiness
The Intersectionality of Identity, Culture & the Rare Disease Experience — Health Equity Forum 2023
This panel spoke during the 2023 Health Equity Forum in San Diego, California. In order to build […]
Read moreKeynote Address: Racism – A Historical Foundation for Systemic Disparities — Health Equity Forum 2023
This keynote address kicked off the 2023 RARE Health Equity Forum. Racial inequities persist in all of […]
Read moreEngagement Strategies How to Find and Leverage Partnerships with Community Leaders — Health Equity Forum 2023
This panel was part of the 2023 Health Equity Forum in San Diego, California. Building relationships with […]
Read moreUK Biobank Achieves Milestone with 500,000 Genomes
Rare Daily Staff The UK Biobank released new data from whole genome sequencing 500,000 people, data expected […]
Read moreCreate Your Strategy: The RARE Research Roadmap — 2023 RARE Advocacy Summit
As part of the Becoming a Research Ready Organization track from the 2023 RARE Advocacy Summit, this […]
Read moreResearchers Identify Range of New CRISPR Systems for Potential Gene Editing
Rare Daily Staff Scientists at the Broad Institute of MIT and Harvard, the McGovern Institute for Brain […]
Read moreMental Health & Wellness
Global Genes Team Collaborates with Homocystinuria Community and Researchers on Report
The Homocystinuria (HCU) community, Dr. Kim Chapman (Children’s National Rare Disease Institute), Dr. Mary Chapman (UPMC Children’s […]
Read moreFive Resources For Grieving
August 30 is National Grief Awareness Day. As there are many types of grief and no two […]
Read moreAutoinflammatory Alliance: Bringing Hope and Changing Lives For Those Living With Autoinflammatory Disease
August is Autoinflammatory Disease Awareness Month. One of the more common rare diseases that falls into the […]
Read moreAutoinflammatory Disease: The Invisible Epidemic
August is Autoinflammatory Disease Awareness Month. One of the more common rare diseases that falls into the […]
Read moreImplementing a Decentralized Clinical Trial Design for Children with SCN2A & SCN8A Developmental and Epileptic Encephalopathies (DEEs)
contribution from Praxis Precision Medicines Praxis Precision Medicines is a clinical biopharmaceutical company with the largest portfolio […]
Read moreA Look Back and Forward for Health Equity at the 3rd Annual RARE Health Equity Forum
The 2023 RARE Health Equity Forum will be held in San Diego, California, September 18 and 19. […]
Read moreNavigating the 2023 Global Genes RARE Advocacy Summit: A Choose-Your-Own-Adventure Extravaganza!
Welcome, brave adventurers, to the Global Genes RARE Advocacy Summit! Get ready to embark on a wild […]
Read moreKiora, Looking to Conserve Cash, Sharpens Focus on Rare Retinal Diseases
Rare Daily Staff Kiora Pharmaceuticals said it will sharpen its focus on rare retinal diseases and will […]
Read moreWhy should RARE Disease dads attend the RARE Advocacy Summit during Week in Rare?
1. You will know you are not alone. As a rare disease dad myself (my son Lucas […]
Read moreRARE Daily
Pharvaris Reports Positive Top-line Phase 2 Study Data of Deucrictibant for Treatment of HAE Attacks
Rare Daily Staff Swiss biopharma Pharvaris reported positive top-line data from the CHAPTER-1 phase 2 clinical study […]
Read moreSenator Argues for Novel Payment Mechanisms to Fund Gene Therapies
Rare Daily Staff The growing number of high-priced, potentially curative gene therapies advancing toward a healthcare market […]
Read moreRenagade Therapeutics Cuts 10 Percent of Workforce
Rare Daily Staff Renagade Therapeutics is laying off 10 percent of its staff in order to speed […]
Read moreThe Lancet Publishes Geron’s Phase 3 Results in Lower Risk MDS
Rare Daily Staff Geron said The Lancet published positive results from the IMerge phase 3 trial investigating […]
Read moreFDA Grants Osteal Breakthrough Therapy Designation for Treatment for Periprostehtic Joint Infection
Rare Daily Staff The U.S. Food and Drug Administration granted Breakthrough Therapy designation for VT-X7, Osteal Therapeutics’ […]
Read moreIndustry Group BIO Names Crowley President and CEO
Rare Daily Staff The industry group Biotechnology Innovation Organization has named John Crowley, whose work as a […]
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