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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
What Being Undiagnosed Means in the Rare Community
April 29 is Undiagnosed Disease Day, and Global Genes reached out to the Undiagnosed Disease Network Foundation […]
Read moreSharing Fibrodysplasia Ossificans Progressiva Stories During Global FOP Awareness Day
Global FOP Awareness Day is on April 23, and Global Genes asked Cathryn Roys, Fundraising and Special […]
Read moreJourney to a Diagnosis for Kabuki Syndrome
Kristin was diagnosed with Kabuki syndrome at age 11, and had her diagnosis confirmed with genetic testing […]
Read moreRecognizing the Intersection of Stress and Hope
Shannon Bennett, RARE Concierge Patient Navigator for Global Genes and mother to a daughter diagnosed with a […]
Read moreTo Walk A Mile In My Shoes
These shoes are something special, but could you walk a mile in them? They represent all things […]
Read moreThe Making of MOGAD Awareness Month and the Creation of “The Flock”
Julia Lefelar was diagnosed with MOG (Myelin Oligodendrocyte Glycoprotein) Antibody Disease, also known as MOGAD in 2017, […]
Read moreLatest News
Shundra Wooten on her short film “Keep It Moving”
Shundra Wooten was diagnosed with Spinocerebellar Ataxia 3, and made a short film “Keep It Moving” to […]
Read moreProfluent Releases AI-Created and Open-Source Gene Editor & More — This Week in RARE Daily
This Week in RARE Daily is a feature from Global Genes where you can get a quick […]
Read moreShantel Sonier on “Glitching with Nick”
Shantel Sonier, a rare mom and caregiver to Nick, who was diagnosed with GRIN2A, created the short […]
Read moreLesley Holroyd on her short film “Born to be Heard”
Lesley Holroyd, who lives with congenital adrenal hyperplasia, discusses what she learned about filmmaking and telling her […]
Read more“Miss Diagnosis” breaking the rules with Tara Rule
Daniel DeFabio, Director of Community Engagement for Global Genes, spoke to Tara Rule about what rules should […]
Read moreNational DNA Day: Cracking the Code on Rare Diseases and Unlocking Hope
by Shruti Mitkus, Ph.D. National DNA Day is celebrated each year on April 25th to commemorate the […]
Read moreIndustry News
New BioMarin CEO Prioritizes Three Programs, Axes Four Others
Rare Daily Staff BioMarin Pharmaceutical said that after a strategic R&D asset review to maximize profitability, it […]
Read moreFDA Approves Pfizer’s Hemophilia B Gene Therapy
Rare Daily Staff The U.S. Food and Drug Administration approved Pfizer’s Beqvez, a gene therapy for the […]
Read moreModerna and OpenAI Collaborate to Advance mRNA Medicine
Rare Daily Staff Moderna and OpenAI announced an ongoing collaboration, which will integrate generative AI throughout the […]
Read moreRegeneron and Mammoth Collaborate on Next-Gen CRISPR Gene Editing for Multiple Diseases
Rare Daily Staff Regeneron Pharmaceuticals and Mammoth Biosciences entered a collaboration to research, develop and commercialize in […]
Read moreRett Syndrome Research Trust Partners with Vivalink to Accelerate Research
Rare Daily Staff Digital health technology provider Vivalink entered into a collaboration with the Rett Syndrome Research […]
Read moreLabcorp to Acquire Invitae Assets
Rare Daily Staff Laboratory services company Labcorp said it’s $239 million bid for assets of the medical […]
Read morePerrigo Receives Offer to Divest its Rare Diseases Business for up to $295 Million
Rare Daily Staff Perrigo Company, a provider of consumer self-care products, said that Spanish pharmaceutical company Esteve […]
Read moreAddressing a Shortage of Genetic Counselors with AI
As genetic testing continues to expand, it is bringing into focus a shortage of genetic counselors who […]
Read moreEndeavor Bio Raises $132.5 Million to Advance Pipeline
Rare Daily Staff Endeavor BioMedicines raised $132.5 million in a series C financing, including the conversion of […]
Read moreGenetic Counseling
NEXT Report 2024: Rewriting the Rules
Over the past year, technological advances in rare disease drug and therapy development, coupled with the tenacity of rare disease patients and advocates, have prevailed despite the challenges of financial difficulties in biopharma. Next-generation patient advocates continue to take an active role in drug development, as outlined in the 2024 NEXT Report.
Read moreImportance of Sharing Family History in a Huntington’s Disease Family
by Kathleen Langley I am from a Huntington’s disease (HD) family. I tested negative in the 1990’s, […]
Read moreA 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 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 moreTop 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 moreResearch Readiness
Expanding Access to Genome Sequencing in Rural Populations & More — This Week in RARE Daily
This Week in RARE Daily is a feature from Global Genes where you can get a quick […]
Read moreFinancings of Public Rare Disease Drug Developers Soar & More — This Week in RARE Daily
This Week in RARE Daily is a feature from Global Genes where you can get a quick […]
Read morePromise and Challenges of Gene-Editing and Other Genomic Medicines — 2024 NEXT Report
When scientists completed the Human Genome Project in 2003, it provided, among other things, a reference genome […]
Read moreNCATS Seeks Applicants for Rare Disease Clinical Research Funding & More — This Week in RARE Daily
This Week in RARE Daily is a feature from Global Genes where you can get a quick […]
Read moreNCATS Seeks Applicants for Rare Disease Clinical Research Consortia Funding
Rare Daily Staff The National Institutes of Health’s National Center for Advancing Translational Sciences is seeking new […]
Read moreFeinstein Institutes Secures $6.1 Million NIH Grant for Red Cell Disorder Research
Rare Daily Staff The National Institutes of Health awarded a $6.1 million research grant to the Feinstein […]
Read moreMental Health & Wellness
Two Rare Disease Drug Developers Raise Combined $152.5 Million in PIPE Offerings
Rare Daily Staff Two companies developing therapeutics for rare diseases, Zura Bio and Benitec Biopharma, raised $152.5 […]
Read moreThe Impact of Grants Provided For Ukraine Relief
Healthcare Education Institute was one of six organizations providing aid and support to those affected by the […]
Read moreMental Health Resources for the Rare Disease Community
Those in the rare disease community may experience a variety of mental health issues, ranging from anxiety […]
Read moreCelebrating the Most Expensive Drug for the Disease You Never Heard Of & More — This Week in RARE Daily
This Week in RARE Daily is a feature from Global Genes where you can get a quick […]
Read moreThe Vital Role of Education in Patient Advocacy: A Gene Therapy Perspective
Gene therapy promises to revolutionize medicine, particularly for rare diseases, approximately 80% of which are genetic in […]
Read moreFDA Approves First Gene Therapy for Children with MLD & More — This Week in RARE Daily
This Week in RARE Daily is a feature from Global Genes where you can get a quick […]
Read moreRare, Ultra-Rare & Hyper-Rare: A Search for Paths Forward — 2024 NEXT Report
Over 40 years after the Orphan Drug Act of 1983, the definition of rare disease as it […]
Read moreRecapping Global Genes staff activities during Rare Disease Week
Mackenzie Abramson provides a recap of Rare Disease Day and Rare Disease Week activities in Washington D.C. […]
Read moreQuick and Simple Strategies to Help You and Your Family Reset and Recharge
As a rare disease caregiver or care partner, you may be feeling increasing pressure to do it […]
Read moreRARE Daily
FDA Grants Accelerated Approval to Day One’s Ojemda for Childhood Brain Tumor
Rare Daily Staff The U.S. Food and Drug Administration has granted accelerated approval to Day One Biopharmaceuticals’ […]
Read moreProfluent Releases AI-Created and Open-Source Gene Editor
Rare Daily Staff Profluent said it demonstrated the first successful precision editing of the human genome with […]
Read moreEC Grants Accelerated Approval to Travere and CSL Vifor’s Filspari for Rare Kidney Disease
Rare Daily Staff The European Commission granted accelerated approval to Travere Therapeutics and CSL Vifor’s Filspari for […]
Read moreFDA Approves Novartis’ Lutathera for Children with Rare Neuroendocrine Tumors
Rare Daily Staff The U.S. Food and Drug Administration approved Novartis’ radioligand therapy Lutathera for the treatment […]
Read moreSeamless Raises $25 Million in Seed Financing, Expands to U.S. to Advance Programmable Gene Editing Platform
Rare Daily Staff German biotech Seamless Therapeutics closed a $25 million in seed financing to support the […]
Read moreLifeArc Invests $50 Million to Launch Rare Disease Research Centers
Rare Daily Staff The UK charity LifeArc is investing $50 million (£40 million) to launch four new […]
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