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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
Caregiver Mental Health: We are Renewable Resources with Finite Capacity
May is Mental Health Awareness Month, so Global Genes reached out to mental health professionals in the […]
Read moreBeyond the Diagnosis: The Critical Need for Mental Health Support in the Rare Community
May is Mental Health Awareness Month, so Global Genes reached out to mental health professionals in the […]
Read moreUnveiling the Hidden Heroes: Recognizing Rare Caregivers During Mental Health Awareness Month
May is Mental Health Awareness Month, so Global Genes reached out to mental health professionals in the […]
Read moreThe Road Continues Past Ehlers-Danlos Diagnosis
Almost 10 years ago, Dawn wrote about her diagnosis with Ehlers-Danlos syndrome. We asked her to provide […]
Read moreWhat 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 moreLatest News
FDA Clears First Trial of Prime Medicine’s Editing Therapy & More — This Week in RARE Daily
This Week in RARE Daily is a feature from Global Genes where you can get a quick […]
Read moreShundra 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 moreIndustry News
AstraZeneca Drug Regimen Improves PFS in First-Line Rare Blood Cancer
Rare Daily Staff Positive high-level results from an interim analysis of the ECHO phase 3 trial showed […]
Read moreSoleno Raises $138 Million in Public Offering to Advance Treatment for Prader-Willi
Rare Daily Staff Rare disease focused biotech Soleno Therapeutics raised $138 million in an underwritten public offering […]
Read moreLatus Bio Launches with $54 Million to Deliver Gene Therapies Directly to the Brain
Rare Daily Staff Latus Bio, a company developing improved gene therapies that target central nervous system disorders, […]
Read moreAI Used to Identify Undiagnosed Patients with Immune Disorder through EHRs
Rare Daily Staff Researchers were able to identify people suspected of having an undiagnosed, rare immunodeficency through […]
Read moreFDA Grants Fast Track to Amolyt’s Experimental Hypoparathyroidism Candidate
Rare Daily Staff The U.S. Food and Drug Administration granted Fast Track designation to Amolyt Pharma’s lead […]
Read moreModerna and Metagenomi End Next-Generation In Vivo Gene Editing Collaboration
Rare Daily Staff Moderna and Metagenomi have agreed to terminate their collaboration to develop next-generation, in vivo […]
Read moreUsing Directed Evolution to Develop New Vectors for Genetic Medicines
Much of the challenge of developing genetic medicines lies in having the right vector to deliver the […]
Read moreAtsena Reports Positive Data from First Cohort of Trial Evaluating Gene Therapy for XLRS
Rare Daily Staff Atsena Therapeutics reported positive preliminary data from the first cohort of the ongoing LIGHTHOUSE […]
Read moreCreyon and Cajal Enter Partnership to Develop ASOs for Neurodegenerative Diseases
Rare Daily Staff Creyon Bio and Cajal Neuroscience have entered a partnership to develop novel antisense oligonucleotide […]
Read moreGenetic Counseling
National 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 moreNEXT 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 moreResearch Readiness
Researchers Uncover Genetic Cause of Rare Neurological Disease
Rare Daily Staff A multinational study by scientists at the University of Utah has identified the genetic […]
Read moreExpanding 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 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
Harmony Bio Acquires Epygenix, Adding Late-Stage Rare Epilepsy Assets
Rare Daily Staff Harmony Biosciences has acquired Epygenix Therapeutics, accelerating its growth strategy by adding a rare […]
Read moreMustang Bio Raises $4 Million in Public Offering
Rare Daily Staff Mustang Bio, a company focused on cell and gene therapies for difficult-to-treat cancers and […]
Read moreTeleRare Health Launches National Virtual Clinic for People with Rare Diseases
Rare Daily Staff TeleRare Health launched its service to provide virtual care for people and their families […]
Read moreONO to Acquire Deciphera for $2.4 Billion
Rare Daily Staff Japan-based ONO Pharmaceutical agreed to acquire Deciphera Pharmaceuticals, a company with a portfolio of […]
Read moreFDA Approves X4 Pharma’s Xolremdi for WHIM Syndrome
Rare Daily Staff The U.S. Food and Drug Administration has approved X4 Pharmaceuticals’ Xolremdi capsules for use […]
Read moreFDA Clears First Trial of Prime Editing Therapy
Rare Daily Staff The U.S. Food and Drug Administration has granted clearance to Prime Medicine to begin […]
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