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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
From Rare Mom & Caregiver to Champ1 Advocate
Richelle Wissink is a rare mom and caregiver and became an advocate after her son was diagnosed […]
Read moreBig Boost in Rare Disease Financings & More — This Week in RARE Daily
This Week in RARE Daily is a new feature from Global Genes where you can get a […]
Read moreGiving Thanks during Trisomy Awareness Month
March is Trisomy Awareness Month, and Global Genes asked Gary David, a rare disease dad, caregiver, and […]
Read moreJourney of a Ph.D. with Muscular Dystrophy
I’m a 35 year old with Muscular Dystrophy from the Land of Enchantment (aka New Mexico). As […]
Read moreA Survivor: Diagnosed with Tetralogy of Fallot at birth, then DiGeorge syndrome
Michelle Padilla was born with a congenital heart defect, Tetralogy of Fallot, and was later diagnosed with […]
Read moreBeing a caregiver for my dad who is living with the same disease I will get one day
Erin Paterson is a caregiver for her father, who lives with Huntington’s disease, and has tested positive […]
Read moreLatest News
Rare, 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 moreSigns of Depression and Anxiety in Children and Teens Living with a Rare Condition
Kids with rare diseases experience depression and anxiety at 2-3 times a higher rate than their peers. […]
Read moreFinding Support: A Compilation of Emotional and Mental Health Resources in the United States
There are a number of mental health resources available for rare disease patients, families and caregivers, but […]
Read moreGetting Help & Finding the Right Fit: Available Emotional and Mental Health Resources
Many resources are available for mental health care, but it is important to carefully research them, access […]
Read moreIndustry News
Equity, Diversity & Inclusion Seminar: Defining Your Underserved & Underrepresented Patient Population
Global Advocacy Alliance Lunch & Learn Equity Diversity & Inclusion Seminar, Part 1 Defining Your Underserved & […]
Read moreAstraZeneca to Acquire Amolyt Pharma
AstraZeneca to Acquire Amolyt Pharma for Rare Endocrine Disease Portfolio Rare Daily Staff AstraZeneca will acquire Amolyt […]
Read moreSpruce Cuts Staff as CAH Study Misses Primary Endpoint
Rare Daily Staff Spruce Biosciences said topline results from its phase 2b study of tildacerfont in adult […]
Read moreFDA Awards Keros Fast Track Designation for Experimental Therapy to Treat MDS
Rare Daily Staff The U.S. Food and Drug Administration has granted Fast Track designation for Keros Therapeutics’ […]
Read moreFDA Expands Use of Mirum’s Livmarli to Include PFIC
Rare Daily Staff The U.S. Food and Drug Administration has expanded the approval of Mirum Pharmaceuticals’ Livmarli […]
Read moreHelping People with Undiagnosed Rare Diseases Find Answers
Michele Herndon’s son Mitchell began developing symptoms of an ultra-rare neurological condition in 2012. He went for […]
Read moreResearchers Take Aim at DMD Therapies to Make Case for Timely Completion of Confirmatory Trials
Rare Daily Staff Spending on targeted DMD therapies reached $3.7 billion from 2016 to 2022, despite limited […]
Read moreAs Spending on Cell and Gene Therapies Grows, Challenges Remain
Rare Daily Staff Spending on cell and gene therapies grew to $5.9 billion in 2023, a 38 […]
Read moreTakeda Reports Positive Topline Results from Phase 2 Study in ITP
Rare Daily Staff Takeda reported positive topline results from a phase 2, randomized, double-blind, placebo-controlled study evaluating […]
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
Global Genes’ Mackenzie Abramson continues her Rare Disease Week adventures in Washington D.C.
Mackenzie Abramson, Senior Manager of Research Program Communications for Global Genes, Rare Patient and Advocate, takes on […]
Read moreGlobal Genes’ Mackenzie Abramson takes on Washington D.C. at Rare Disease Week
Mackenzie Abramson, Senior Manager of Research Program Communications for Global Genes, Rare Patient and Advocate, takes […]
Read moreRAREly Told Stories Workshop
Have you wanted to tell your rare disease story as a documentary or a short video? Global […]
Read moreBeginner’s Guide to Community Activation — RARE Advocacy Summit 2023
As part of the Community & Capacity Building track from the 2023 RARE Advocacy Summit, this panel […]
Read moreSpinal Muscular Atrophy Case Study — 2023 RARE Advocacy Summit
As part of the Rare Disease Master Class Science & Tech Innovation track from the 2023 RARE […]
Read morePower of Partnering: Rosamund Stone Zander Translational Research Neuroscience Center — 2023 RARE Advocacy Summit
As part of the Becoming a Research Ready Organization track from the 2023 RARE Advocacy Summit, this […]
Read moreMental Health & Wellness
Depression and Anxiety: Understanding the Signs and Symptoms and Getting Help
Having a chronic and rare disease is a challenging experience, and especially difficult for children and adolescents, […]
Read moreAddressing and Managing Pain: Resources for Patients Who Have Pain
The management of chronic pain has proven to be a significant challenge for rare diseases patients and […]
Read moreCIRM Awards $56 Million to Advance Clinical Research, Including Lupus and Glioblastoma
CIRM Awards $56 Million to Advance Clinical Research, Including Lupus and Glioblastoma Rare Daily Staff The California […]
Read moreLove is in the RARE – Tips for Love from the Rare Disease Community
Valentine’s Day celebrates romantic love, but how does rare disease throw a curve into typical loving relationships? […]
Read moreIn the Realm of Rarity: Joshua Salisbury, first place winner of 2023 Cox Scholarship
Read moreListen Louder: Rahi Patel, second place winner of 2023 Cox Scholarship
Read moreHANDling Your Story: Tips For Telling Your Story
by Mary Morlino RARE Patient & Patient Advocate I created this HANDling Your Story tip sheet because […]
Read moreLetter from Charlene Son Rigby, CEO to Kick Off 2024
by Charlene Son-Rigby CEO, Global Genes The past year was a significant one for Global Genes, marked […]
Read moreA Listening Ear Can Diagnose and Heal: Stacey Sklepinski, third place winner of 2023 Cox Scholarship
Read moreRARE Daily
Almirall Licenses Eloxx Pharma’s ZKN-013 for Rare Dermatological Diseases
Rare Daily Staff Spanish biopharma Almirall entered an exclusive license agreement with Eloxx Pharmaceuticals to develop and […]
Read moreFDA Grants Rare Pediatric Disease Designation to PepGen’s Therapeutic for DMD
Rare Daily Staff The U.S. Food and Drug Administration granted both orphan drug and rare pediatric disease […]
Read moreBioMarin to Present Long-Term Data on Achondroplasia Drug, New Data on Hypochondroplasia
Rare Daily Staff BioMarin said researchers will present positive results from an investigator-sponsored study of its drug […]
Read moreAllogene and Arbor Enter Gene Editing Licensing Deal for Allo CAR T Platform in Autoimmune Disease
Rare Daily Staff Allogene Therapeutics and Arbor Biotechnologies entered a non-exclusive, global gene editing licensing agreement for […]
Read moreRegulus Raises $100 Million in PIPE Financing to Advance Rare Kidney Disease Pipeline
Rare Daily Staff Regulus Therapeutics, a company focused on innovative microRNA therapeutics for orphan kidney diseases, entered […]
Read moreIncyte Reports Positive Data from Phase 2 Study in Rare Skin Disease
Rare Daily Staff Incyte reported results that its phase 2 study evaluating the efficacy and safety of […]
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