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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
Discovering 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 moreThe Continuous Grief of Chronic Illness
by Megan Marjorie “I should be happy” I repeated to myself in my head. I kept on […]
Read moreFrom rare disease diagnosis to nonprofit advocacy organization – The Fairy Goddess Mother Project
In March of 2022, at age 47, I was diagnosed with Vulvar Lichen Sclerosus (VLS), an autoimmune […]
Read moreMaryland Establishes Rare Disease Advisory Council through SB188, HB302
by Mary Morlino Founder, MarylandRARE MarylandRARE is thrilled to announce the signing of Senate Bill 188 (SB […]
Read moreWe Are Brave Together Is Passionate About the Mental Health of Caregivers
By Jessica Patay Founder/Executive Director, We Are Brave Together May is a time to honor mothers everywhere […]
Read moreWishing doctors looked at the whole picture when it comes to healthcare
by Megan Marjorie Embarrassment sank deeper with every syllable — as In pure frustration. I dumped the […]
Read moreLatest News
The RARE Research Roadmap – 2023 RARE Drug Development Symposium
Read moreMental Health in Rare Disease: Taking Care of Your Mind Is Taking Care of Your Body
May is Mental Health Awareness Month. Taking care of your mental health is key, whether you are […]
Read moreHow Can the FDA Improve Processes for Rare Diseases? – 2023 RARE Drug Development Symposium
Read moreThe RARE Research Roadmap – 2023 RARE Drug Development Symposium
Read moreThe Regulatory Landscape: Pathways, End Points, and Clinical Trials – 2023 RARE Drug Development Symposium
Read moreIntellectual Property: Balancing Stakeholders’ Rights – 2023 RARE Drug Development Symposium
Read moreIndustry News
Ultragenyx and Mereo Report Positive Data in Phase 2/3 Study in Rare Bone Disease
Rare Daily Staff Ultragenyx Pharmaceutical and Mereo BioPharma Group reported positive data from the dose-selection phase 2 […]
Read moreAlkeus Raises $150 Million to Advance Treatment for Stargardt Disease to Market
Rare Daily Staff Alkeus Pharmaceuticals said it has raised a $150 million in a series B financing […]
Read moreSynlogic Cuts 21 Percent of Staff as It Initiates Phase 3 Study in PKU
Rare Daily Staff Synlogic said it was cutting 21 percent of its staff to prioritize resources to […]
Read moreTakeda Ends Agreement with Poseida
Poseida Therapeutics, in a filing with the U.S. Securities and Exchange Commission, said Takeda had terminated its […]
Read moreModulo Bio Launches to Develop Treatments for Neurodegenerative Disease Treatments
Rare Daily Staff Modulo Bio emerged from stealth mode with $8 million in seed funding that it […]
Read moreNew Zealand Patient Advocacy Group Calls for Newborn Screening to Include SMA
Rare Daily Staff Rare Disorders NZ, a national rare disease patient advocacy group in New Zealand, has […]
Read moreFDA Grants Fast Track Designation to Arcturus’ mRNA Therapeutic Candidate for OTC
Rare Daily Staff The U.S. Food and Drug Administration has granted Fast Track designation to Arcturus Therapeutics’ […]
Read morePharming Sells Priority Review Voucher for Pre-Set Price of $22.1 Million
Rare Daily Staff Pharming Group said it has entered into a definitive agreement with Novartis Pharma to […]
Read moreFDA Grants Fast Track Designation for Inhibrx Treatment of Alpha-1 Antitrypsin Deficiency
Rare Daily Staff The U.S. Food and Drug Administration granted Fast Track designation to Inhibrx’ INBRX-101, an […]
Read moreGenetic Counseling
CEO 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 moreUnderdiagnosed? Fight For a More Complete Diagnosis
by Effie Parks Note: this article first appeared on Global Genes’ RARE Portal You or a loved […]
Read moreAnatomy of a Next Generation Rare Disease Patient Advocate
Patience is not always a virtue. In the world of rare disease, drug developers and patient advocates […]
Read moreGlobal Genes Impact Report: Building on a Successful 2022
To our community members: The year 2022 was a significant one for the rare disease community as […]
Read moreSisters with Sanfilippo Syndrome
by Anna Kidwell I am mom to two girls, Ashleigh and Brooke, who were diagnosed with Mucopolysaccharidosis […]
Read moreResearch Readiness
Bringing Precision to CRISPR-Based Genome Editing
CRISPR genome editing has the potential to revolutionize the treatment of diseases, but the imprecision of its […]
Read moreNew Technologies in Early Stage Research – 2023 RARE Drug Development Symposium
Read moreUnderstanding Data for Basic Research – 2023 RARE Drug Development Symposium
Read moreCharting the Path to Treatments – 2023 RARE Drug Development Symposium
Read moreStudy Finds Rare Disease Experts Support DNA Sequencing in Newborns
Rare Daily Staff Routine newborn screening will test infants for up to 60 treatable diseases, but even […]
Read moreNIH Researchers Uncover New Details on Rare Immune Disease
Rare Daily Staff In an 11-year study, researchers at the National Institutes of Health have further characterized […]
Read moreMental Health & Wellness
2023 Health Equity in RARE Impact Grant Recipients Announced
Nine Global Advocacy Alliance organizations received the 2023 Health Equity in RARE Grants. These grants will provide […]
Read moreFunction Oncology Launches with $28 Million to Advance CRISPR-powered Personalized Functional Genomics Platform
Rare Daily Staff Function Oncology, a precision medicine company advancing a CRISPR-powered personalized functional genomics platform to […]
Read more2023 RARE Mental Health Grant Recipients Announced
Global Genes is pleased to announce the Rare Disease Patient Advocacy Organizations receiving 2023 RARE Mental Health […]
Read moreSeeking a Sustainable Business Developing on N-of-1 Therapies
The ability to pinpoint the underlying genetic causes of diseases and rapidly generate genetic medicines to address […]
Read moreGlobal Genes Team Shares Their Stories on World Rare Disease Day
My daughter Juno was diagnosed with a very rare, severe neurodevelopmental condition called STXBP1-related disorder six years […]
Read moreRARE Global Advocacy Alliance Member Spotlight: Geoffrey Gee
Read moreRare Disease Caregiver Resources
National Caregiver Day in the United States falls on the third Friday of February every year, on […]
Read moreFinding Me Through This Rare Journey
Patient or Advocate: Brittany Anderson Disease Affiliation: Fibrous Dysplasia, McCune Albright Syndrome, Panhypopituitarism, Adrenal Insufficiency, Trigeminal Neuralgia, […]
Read moreBorn to be rare, born to fight for tomorrow
Patient or Advocate: Nick Irwin Disease Affiliation: Porencephaly Hi. My name is Nick. I’m 6 foot 3 […]
Read moreRARE Daily
Belite Bio Raises $30 Million in Public Offering
Rare Daily Staff Belite Bio, a clinical stage company focused on advancing novel therapeutics targeting retinal degenerative […]
Read moreEMA Grants PRIME Designation to Rocket’s Gene Therapy for Danon Disease
Rare Daily Staff The European Medicines Agency granted Priority Medicines (PRIME) designation to Rocket Pharmaceutical’s RP-A501, the […]
Read moreFDA Grants Priority Review to BMS for Treatment for Rare Form of NSCLC
Rare Daily Staff The U.S. Food and Drug Administration granted priority review to Bristol Myers Squibb’s repotrectinib, […]
Read morePfizer Reports Positive Marstacimab Results from Pivotal Phase 3 Hemophilia A and B Trial
Rare Daily Staff Pfizer reported that the pivotal phase 3 BASIS clinical trial evaluating marstacimab has met […]
Read moreFDA Places Clinical Hold on PepGen’s Application to Initiate a Phase 1 Study in DM1
Rare Daily Staff The U.S. Food and Drug Administration notified PepGen’s that it was placing a clinical […]
Read moreBioLight Enters Agreement with Alexion to Explore Technology To Diagnose Retinal Diseases
Rare Daily Staff Israeli ocular disease focused biotech BioLight signed a research collaboration agreement with Alexion, AstraZeneca […]
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