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RARE Daily: the official Global Genes blog
Browse the latest news, articles, and blog posts from Global Genes.
Featured
Spotting Neuromuscular Disease Red Flags
Many older adults may dismiss dropping objects, struggling with stairs, or tiring on short walks as a […]
Read moreExpanding into a Global Rare Disease Player through Deal-Driven Innovation
Chiesi Global Rare Diseases has rapidly evolved from a small, regional rare‑disease business into a global organization, […]
Read moreWhen Geography, Cost, and Policy Become as Life-Limiting as a Disease
When Tom Sayiner was diagnosed with the fatal neurodegenerative disease ALS, he and his wife, Tamara, learned […]
Read moreA One-Time Cell Therapy to Reset the Immune System in Autoimmune Diseases
People with rare, severe autoimmune diseases often live for years with progressive, disabling conditions managed by chronic […]
Read moreDrug Development
BioMarin Discontinues Development of Therapy for ENPP1 Deficiency
Rare Daily Staff BioMarin Pharmaceutical said it will discontinue development of its experimental enzyme replacement therapy for […]
Read moreFDA Approves Takeda’s First-in-Class Narcolepsy Type 1 Drug
Rare Daily Staff The U.S. Food and Drug Administration has approved Takeda’s Orzeyful, the first medicine designed […]
Read moreRegulators Align on AMO Pharma Pivotal cDM1 Study
Rare Daily Staff AMO Pharma said regulators in the United States, United Kingdom, and Canada have agreed […]
Read moreOtsuka Reports “Unprecedented” Results for IgA Nephropathy Drug
Rare Daily Staff Otsuka reported positive Phase 3 results for Voyxact that suggest the drug may slow […]
Read moreCOMBINEDBrain-Led Initiative to Evaluate RNA-Based Therapeutic Platform for Neurodevelopmental Conditions
Rare Daily Staff A coalition of rare disease organizations is backing a new research effort at Johns […]
Read moreFDA Advisory Committee Votes Against Effectiveness of Capricor’s Duchenne Cell Therapy
Rare Daily Staff A U.S. Food and Drug Administration advisory committee voted against the effectiveness of Capricor […]
Read moreFDA Grants HAYA’s nHCM Therapy Fast Track Designation
Rare Daily Staff The U.S. Food and Drug Administration granted HAYA Therapeutics Fast Track designation for its […]
Read moreCHMP Issues Negative Opinion on Zevra’s NPC Therapy
Rare Daily Staff European regulators have issued a negative opinion on Zevra Therapeutics’ marketing application for arimoclomol, […]
Read moreJ&J Report Positive Results from Combination of Multiple Myeloma Immunotherapies
Rare Daily Staff Johnson & Johnson said Thursday that an experimental combination of its multiple myeloma drugs […]
Read moreFinance
ARPA-H Makes $27.7 Million Grant to IGI Led Effort to Deliver CRISPR-Based Therapies for IEIs
Rare Daily Staff The Advanced Research Projects Agency for Health has awarded up to $27.7 million to […]
Read moreRoyalty Pharma Acquires Interest in ATTR-CM Therapy for up to $425 million
Rare Daily Staff Royalty Pharma said that it has acquired a portion of Neurimmune’s royalty interest in […]
Read moreRegenxbio Prices $100 Million Public Offering to Bolster Capital Position
Rare Daily Staff Regenxbio has priced an underwritten public offering expected to generate approximately $100 million in […]
Read moreMighty Therapeutics Secures Financing for Up to $150 Million
Rare Daily Staff Mighty Therapeutics, formerly known as Stealth BioTherapeutics, said it has secured up to $150 […]
Read moreARPA-H Awards up to $160 Million to Advance Personalized Curative Medicines for Rare Genetic Diseases
Rare Daily Staff The Advanced Research Projects Agency for Health has awarded up to $160 million in […]
Read moreDeveloper Makes $10 Million Gift to Penn for Preclinical Development of RVCL Therapy
Rare Daily Staff The Clayco Foundation, the philanthropic arm of the Chicago-based real estate development and construction […]
Read moreCIRM Issues $60 Million in Grants Including Funding for FOXG1 Gene Therapy
Rare Daily Staff California Institute of Regenerative Medicine, the state’s stem cell agency, is issuing $60 million […]
Read moreUniQure Raises $225 Million to Back Accelerated Approval Bid for Huntington’s Gene Therapy
Rare Daily Staff UniQure is capitalizing on an improved regulatory outlook for its experimental Huntington’s disease gene […]
Read moreSangamo Files for Bankruptcy, Lilly and Astellas Line up as Lead Bidders for Assets
Rare Daily Staff Sangamo Therapeutics has filed for Chapter 11 bankruptcy protection and entered into asset sale […]
Read moreMore Stories
Scientists Uncover New Autoimmune Disease Genes
Rare Daily Staff Researchers have uncovered new genetic clues behind autoimmune diseases by looking at how DNA […]
Read moreBioMarin Enters Research Collaboration with n-Lorem to Develop First-in-Disease Medicine
Rare Daily Staff BioMarin Pharmaceutical and the n-Lorem Foundation have entered an early-stage research collaboration aimed at […]
Read moreJett Foundation Launches $10.7 Million Campaign to Build Duchenne-Focused Retreat
Rare Daily Staff Jett Foundation has launched a $10.7 million capital campaign to build Jett’s Place, a […]
Read moreSarepta Names Former AbbVie Exec as CEO
Rare Daily Staff Sarepta Therapeutics has appointed veteran biopharmaceutical executive Michael Severino as CEO, replacing Doug Ingram, […]
Read moreCommission Calls for National Strategy on Rare Disease Drug Development
Rare Daily Staff A bipartisan legislative advisory commission is calling for a unified federal strategy to speed […]
Read moreNewly Launched Raremap Helps Rare Disease Community Navigate UK Ecosystem
Rare Daily Staff LifeArc, the self-funded UK organization focused on addressing high-risk and complex medical challenges, has […]
Read moreWhat’s Happening
Acting FDA commissioner pledges to prevent political interference in rare disease meeting
Global Genes was honored to be invited by the FDA to participate in a closed roundtable discussion […]
Read moreCURE SYNGAP1 announces $111,960 investment in RARE-X partnership to advance ProMMIS patient-reported outcome measure data collection – PR46
CURE SYNGAP1 is proud to announce a strategic investment of $111,960 in RARE-X to accelerate the collection of high-quality […]
Read moreSavara’s Early Access Program
Savara’s Early Access Program (EAP) for molgramostim inhalation solution (molgramostim) in Autoimmune Pulmonary Alveolar Proteinosis (Autoimmune […]
Read morePublication Announcement — RARE-X: Advancing Rare Disease Research Through Patient-Driven Data
Global Genes is excited to announce “RARE-X: A patient-driven approach for collecting symptom and patient-reported outcome data […]
Read moreAccelerating Rare Disease Research Through Collaboration: Global Genes and Notre Dame Launch the Research Acceleration Program
Global Genes and University of Notre Dame have partnered to launch the Research Acceleration Program, a collaborative […]
Read moreRARE Advocacy Exchange Session 8, Knowing Your Rare Rights
This session recording is from Oct. 16, 2025 Rare Advocacy Exchange Session 8: Knowing the Rights for […]
Read moreGlobal Genes Guide to Starting a Nonprofit Patient Advocacy Organization
This Global Genes Quick Guide is a resource for advocates focused on pressing topics causing challenges in […]
Read moreRARE Advocacy Exchange Session 7, Getting a Precise Genetic Diagnosis
This session recording is from Sept. 18, 2025 Rare Advocacy Exchange Session 7: Getting A Precise Diagnosis […]
Read moreRARE Advocacy Exchange Session 6 : Grieving a RARE Diagnosis
Rare disease grief differs from the grief we commonly associate with losing a loved one to death. […]
Read moreReports
Early and Often: Reimagining patient community engagement to improve clinical trials feasibility
The Global Genes Corporate Alliance has developed a new white paper that shows the transformative role of […]
Read moreContinuing Advocacy and Expanding Research Efforts: Global Genes 2023 Impact Report
To our community members: For both the rare disease community and Global Genes, 2023 marked a year […]
Read moreGlobal Genes 2023 RARE Impact Grants Report
Over the past decade, Global Genes’ RARE Impact Grant Program has provided grants to rare disease patient […]
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