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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 moreLatest
An Ultra-Rare Disease Drug Developer Tries to Navigate Regulatory Uncertainty
When Stealth Biotherapeutics sought approval for its experimental therapy to treat the ultra-rare and life-threatening condition Barth […]
Read moreCIRM Awards Immusoft $8 Million for MPS I Clinical Program
Rare Daily Staff The California Institute for Regenerative Medicine awarded an $8 million grant to Immusoft of […]
Read moreSenti Bio Prioritizes Pipeline to Extend Cash Runway
Rare Daily Staff Senti Biosciences, which is developing next-generation cell and gene therapies using its proprietary Gene […]
Read moreFDA Grants Fast Track Designation to Syros’ Tamibarotene for Treatment Group of Rare Cancers
Rare Daily Staff The U.S. Food and Drug Administration granted Fast Track designation to Syros Pharmaceuticals’ tamibarotene […]
Read moreMagenta Pauses Dose Escalation Trial of Conditioning Agent Following Patient Death
Rare Daily Staff Magenta Therapeutics said it voluntarily paused a dose escalation trial of its conditioning agent […]
Read moreEMA Amends Policy of Orphan Designations of Therapies for Inherited Retinal Dystrophies
Rare Daily Staff The European Medicines Agency’s Committee on Orphan Medicinal Products said it has amended its […]
Read moreA New, More Inclusive Membership Model for Global Advocacy Alliance Members
Eligibility for Global Genes grants Access to dedicated GAA member meetings at the Annual Summit and other […]
Read moreTalking Can Be Excellent Medicine
Throughout my undergraduate career, I was a camp counselor for Muscular Dystrophy Association’s summer camp. For one […]
Read moreMedical Dignity at Every Turn: Insights Learned from Rare Disease
On reflection, rare disease has always felt a bit personal to me. When my mother was diagnosed […]
Read morePatient Stories
Sinergia Informative
Patient or Advocate: Wendy Gaviria Disease Affiliation: Cartílago de pelo hipo plástico Author: Wendy Gaviria
Read moreCare Tactics: Hoping for a better life for my son
Read moreSupporting Caregiver Decision-Making
Read moreAdvocating for You, the Caregiver
Read moreCreating Care for the Caregiver: Five Minutes at a Time
Read moreThough she be but little, she is FIERCE
Read moreGenetic Counseling
Make a Plan: How to Talk to Your Family
Tips on how to start a conversation, questions to ask, and why learning about your family health history is important.
Read moreMedical Tests to Find Answers
Learn about medical tests that can help explain why certain conditions run in your family.
Read moreHow a Genetic Counselor Can Help You
Genetic counselors can answer questions, explain hard information, and help you find ways to pay for tests.
Read moreMaking Informed and Shared Decisions About Genetic Testing and Clinical Trial Participation
Introduction When healthcare providers work together with patients and caregivers to make decisions about tests, treatments, and […]
Read moreHighlights from 2020 RARE Drug Development Symposium
Read moreRepurposed Drug Saves Life of Child with Rare Lymphatic Disorder
Rare Daily Staff A team of specialists at Children’s Hospital of Philadelphia demonstrated the potential to screen […]
Read moreResearch Readiness
Homology Prioritizes Pipeline to Extend Runway, Moves Focus to PKU Gene Editing Trial
Rare Daily Staff Homology Medicines, in its second quarter earnings report and business update, said it was […]
Read moreProQR Winds Down Clinical Trials to Focus Exclusively on Axiomer RNA-editing Technology
Rare Daily Staff ProQR Therapeutics, following the European Medicines Agency recommendation that the company conduct an additional […]
Read moreNeuroSense Joins EverythingALS Open Innovation Consortium
Rare Daily Staff NeuroSense Therapeutics is joining the EverythingALS Open Innovation Forum, which consists of patients, caregivers, […]
Read moreStem Cell Transplantation Shows Promise in AL Amyloidosis
Rare Daily Staff Researchers report that for highly selected patients, treatment including high-dose chemotherapy and autologous stem […]
Read moreAmylyx Enters Partnership Agreement with Sunnybrook Research to Find New Treatment for ALS
Rare Daily Staff Amylyx Pharmaceuticals said it entered into a two-year, sponsored research agreement with Sunnybrook Research […]
Read moreHelping Hands for GAND Hosts 3rd Biennial Gathering & Scientific Conference
Conference Aims to Bring Together GAND Community to Discuss Latest Advancements, Research and Scientific Progress
Read moreMental Health & Wellness
A New, More Inclusive Membership Model for Global Advocacy Alliance Members
Eligibility for Global Genes grants Access to dedicated GAA member meetings at the Annual Summit and other […]
Read moreRARE Global Advocacy Alliance Member Spotlight: Sonny Mullen
Read moreRare Diseases: Reconciling Uncertainty and Family; Leah Sarah Peer, Medical Student and third place winner of 2022 Cox Scholarship
Read moreTalking Can Be Excellent Medicine; Jenna Kwiecinski, Medical Student and second place winner of 2022 Cox Scholarship
Read moreMedical Dignity at Every Turn: Insights Learned from Rare Disease; Raymond So, Medical Student and first place winner of 2022 Cox Scholarship
Read moreRARE Global Advocacy Alliance Member Spotlight: Giovi Moschoudis
Read moreRARE Global Advocacy Alliance Member Spotlight: David Simon Ross
Read moreWhat We Learned at RARE Health Equity Summit
Read moreRARE Global Advocacy Alliance Member Spotlight: Christopher Velona
Read moreRARE Daily
CIRM Awards Immusoft $8 Million for MPS I Clinical Program
Rare Daily Staff The California Institute for Regenerative Medicine awarded an $8 million grant to Immusoft of […]
Read moreSenti Bio Prioritizes Pipeline to Extend Cash Runway
Rare Daily Staff Senti Biosciences, which is developing next-generation cell and gene therapies using its proprietary Gene […]
Read moreFDA Grants Fast Track Designation to Syros’ Tamibarotene for Treatment Group of Rare Cancers
Rare Daily Staff The U.S. Food and Drug Administration granted Fast Track designation to Syros Pharmaceuticals’ tamibarotene […]
Read moreMagenta Pauses Dose Escalation Trial of Conditioning Agent Following Patient Death
Rare Daily Staff Magenta Therapeutics said it voluntarily paused a dose escalation trial of its conditioning agent […]
Read moreEMA Amends Policy of Orphan Designations of Therapies for Inherited Retinal Dystrophies
Rare Daily Staff The European Medicines Agency’s Committee on Orphan Medicinal Products said it has amended its […]
Read moreTalking Can Be Excellent Medicine
Throughout my undergraduate career, I was a camp counselor for Muscular Dystrophy Association’s summer camp. For one […]
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