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RARE Daily: the official Global Genes blog
Browse the latest news, articles, and blog posts from Global Genes.
Featured
A Small Molecule Therapy to Regenerate Muscle in People with DMD
Duchenne muscular dystrophy is an inherited disease caused by genetic mutations that no longer allow the dystrophin […]
Read moreForging a Faster Path for Gene Therapies
The Bespoke Gene Therapy Consortium, a public-private partnership backed by the Foundation for the National Institutes of […]
Read moreDetermining the Value of Rare Disease Therapies
The small patient populations of rare diseases, the limited natural history of these conditions, and the lack […]
Read moreFinancings of Public Rare Disease Drug Developers Soar in First Quarter 2024
Companies developing drugs for rare diseases raised substantial new capital so far this year, as rare disease […]
Read moreDrug Development
Argenx Highlights gMG Drug Data in CIDP
Rare Daily Staff Argenx said that positive data from its phase 3 ADHERE trial evaluating Vyvgart Hytrulo […]
Read moreFDA Grants Fast Track Designation for Lexeo’s Gene Therapy for Friedreich’s Ataxia Cardiomyopathy
Rare Daily Staff The U.S. Food and Drug Administration has granted Fast Track designation to Lexeo Therapeutics’ […]
Read moreA 37-Year Path to a Diagnose, but Now a Drug May Be Two Weeks Away
It took Courtney Ampezzan 37 years to get a correct diagnosis for her condition, an unusual length […]
Read moreUltragenyx Reports Positive Interim Phase 1/2 Data in Patients with Angelman Syndrome
Rare Daily Staff Ultragenyx Pharmaceutical reported positive new data from the phase 1/2 study of GTX-102 for […]
Read moreBrainStorm Reaches Agreement with FDA on Special Protocol Assessment for ALS Trial
Rare Daily Staff BrainStorm Cell Therapeutics said it has reached an agreement with the U.S. Food and […]
Read moreAmylyx’s Withdrawn ALS Drug Shows Promise in Wolfram Syndrome
Rare Daily Staff Amylyx Pharmaceuticals reported positive interim data from the ongoing phase 2 HELIOS clinical trial […]
Read moreUK MHRA Approves Nulibry to Treat Adult Patients with MoCD Type A
Rare Daily Staff The UK Medicines and Healthcare products Regulatory Agency approved BridgeBio subsidiary Origin Biosciences’ Nulibry […]
Read moreFDA Says It Will Review Stealth’s Application for Barth Syndrome Treatment
Rare Daily Staff The U.S. Food and Drug Administration said it will review Stealth BioTherapeutics’ New Drug […]
Read moreIonis Reports Positive Results from Phase 3 Balance Study of Olezarsen for FCS
Rare Daily Staff Ionis Pharmaceuticals reported full results from its phase 3 Balance study of its lead […]
Read moreFinance
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 moreJ&J Enter Collaborations with Rallybio, Makes Equity Investment
Rallybio said it has entered into a collaboration with Johnson & Johnson to support the development of […]
Read moreZevra Refinances Existing Debt with Up to $100 Million Under New Credit Facility
Rare Daily Staff Rare diseases therapeutics company Zevra Therapeutics said it has entered into a new credit […]
Read moreNvelop Launches with Dual Platforms for In Vivo Delivery of NextGen Genetic Medicines
Rare Daily Staff Nvelop Therapeutics formally announced its launch with $100 million in funding to develop programmable, […]
Read moreFinancings of Public Rare Disease Drug Developers Soar in First Quarter 2024
Companies developing drugs for rare diseases raised substantial new capital so far this year, as rare disease […]
Read moreAeovian Raises $50 Million, Doses First Cohort in Phase 1 TSC Study
Rare Daily Staff Aeovian Pharmaceuticals, a clinical-stage company developing therapeutics that potently inhibit the mTORC1 pathway, completed […]
Read moreCF Foundation Invests Up to $8.5 Million in SpliSense
Rare Daily Staff The Cystic Fibrosis Foundation said it is investing up to $8.5 million in additional […]
Read moreDiagonal Launches with $128 Million to Pioneer Agonist Antibodies to Tackle Rare Diseases
Rare Daily Staff Diagonal Therapeutics, which is pioneering a new approach to discovering and developing agonist antibodies, […]
Read moreRhythm Secures $150 Million in Convertible Preferred Stock Financing
Rare Daily Staff Rhythm Pharmaceuticals has signed an investment agreement with current shareholders, led by Perceptive Advisors […]
Read moreMore Stories
Expanding Access to Genome Sequencing in Rural Populations
Rare Daily Staff Patients in rural area often face challenges accesses cutting-edge medical technologies, such as state-of-the-art […]
Read moreGroup Calls for Use of Genome Sequencing as First Line Test for Patients Suspected of Genetic Disease
Rare Daily Staff The Medical Genome Initiative, a group made up of sequencing giant Illumina and leading […]
Read moreAcross Healthcare Enters Collaboration with n-Lorem Foundation to Develop Submission Platform for Docs
Rare Daily Staff The nonprofit foundation n-Lorem has entered into a collaboration with the technology company Across […]
Read moreHarmony and BioProjet License Potential Treatment for Sleep and Wake Disorders
Rare Daily Staff Harmony Biosciences entered an exclusive licensing agreement with French biotech Bioprojet to develop, manufacture, […]
Read moreVertex Agrees to Acquire Alpine Immune Sciences for $4.9 billion
Rare Daily Staff Vertex Pharmaceuticals said it agreed to acquire immunotherapy developer Alpine Immune Sciences for $65 […]
Read moreGreat Ormond Street Hospital Devises Plan to Expand Access to Gene Therapies
Rare Daily Staff Great Ormond Street Hospital is piloting a new approach to expand access to gene […]
Read moreWhat’s Happening
Expanding 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 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 moreReports
Global Genes 2023 RARE Impact Grants Report
Over the past decade, Global Genes’ RARE Impact Grant Program has provided grants to rare disease patient […]
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 moreGlobal Genes 2022 RARE Impact Grants Report
Over the past decade, Global Genes’ RARE Impact Grant Program has provided funding opportunities for rare patient […]
Read moreMore Resources: Dictionary of Rare Diseases
Search for a specific disease in our extensive list of rare diseases for disease-specific information, news, and resources.
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