News Companies Unite to Support Free Gene Testing in Young Children with Seizures

News Theranexus and BBDF Announce Agreement for Juvenile Batten Disease Treatment Trial

News New Insights into Batten Disease Help Inform Treatments Under Development, Review Says

News Fusion Protein Strategy Creates Trojan Horse for Future Batten Disease Treatment

News Drinking Acidified Water May Have Beneficial Effects in Children With CLN3, Mouse Study Suggests

News Gene Therapy Takes Center Stage at 2019 NORD Summit

News HHS Secretary Alex Azar Touts White House Efforts to Cure Rare Diseases

News Genetic Test Can Provide Definitive Diagnosis of Batten Disease, Canadian Study Suggests

News CLN7 Protein is Involved in Communication Between Nerve and Muscle Cells, Batten Disease Model Shows

News Rare Disease Film Festival Highlights Patient and Researcher Unity

News Fat Molecule May Help Fight CLN3 Disease, Mouse Study Suggests

News Scientists Identify Proteins That May Become Biomarkers of Batten Disease

News Complex Fat Molecule Seen to Aid Lifespan, Ease Neuron Loss in Juvenile Batten Animal Study

News Single Gene Therapy Injection May Stabilize CLN6 Batten Disease in Children, Early Phase 1/2 Data Show

News Gene Therapy Prolongs Lifespan, Alleviates Symptoms of Mice With CLN6 Disease, Study Shows

News Customized Oligonucleotides Treat Seizures in Child with CLN7 Disease in Record Time

News UFC Cheers Young Fan with Batten While Helping to Raise Disease Awareness

News NORD 2019 Rare Disease Summit Set for Oct. 21-22 in Washington, DC

News UK Batten Families Welcome Brineura Access for CLN2 Patients Through NHS

News Taylor’s Tale Co-founder Races to ‘Change the World’ for Batten Disease Community

News Extracellular Vesicles May Enhance Delivery of TPP1 to the Brain for Replacement Therapy in CLN2, Early Study Suggests

News ‘Disorder’ Film Festival Offers Look at Siblings Touched by Rare Diseases

News At NY Genome Center, Legal Expert Presents Ethical Dilemmas in Gene Editing

News Amicus CEO John Crowley Outlines Progress in Batten, Pompe, Fabry Therapies

News Nebraska’s Neena Nizar Seeks Cure for Jansen’s, One of World’s Rarest Diseases

News Neurogene Opens Natural History Study of CLN7 and CLN5 Disease at UT Southwestern

News Oklahoma Ranks Lowest on Programs Key to Rare Diseases on NORD’s 2019 State Report Card

News Rare Disease Groups Seek Public Support to Renew Newborn Screening Act in Senate

News Amicus, Paragon to Collaborate on Gene Therapies for Batten, Other Diseases

News European Initiative Targets Diagnosis, Treatment of Rare Diseases

News Brain-Directed Gene Therapy May Prevent Neurodegeneration in CLN6 Disease, Study Says

News World’s First Alport Stamp Is Macedonian Mom’s Latest Win for Rare Disease Patients

News Motor Impairments Are Early Sign of Juvenile Batten Disease, Study Finds

News NORD Honors Industry, Patient Advocates at Rare Impact Awards Gala

News FDA Grants Fast Track Designation to ABO-202 Gene Therapy for Infantile Batten Disease

News Eurordis Unveils Integrated-care Initiative for Rare Disease Patients

News ‘Rare Barometer’ Program Helps Eurordis Shape EU Rare Disease Policy

News Fycompa May Help Control Involuntary Muscle Jerks in Patients With CLN2 Disease, According to Case Report

News Blood Spot Test May Aid Early Diagnosis of Infantile Batten Disease, Study Says

News Nationwide Children’s Hospital Names Lobby in Honor of Gray Foundation

News Agency Unveils RaDaR to Help Patient Groups Develop Rare Disease Registries

News FDA Clears ABO-202 Gene Therapy Candidate for Clinical Trials in Infantile Batten Disease

News Rare Diseases Constitute a ‘Public Health Issue,’ NCATS Director Warns

News CLN3 Deficiency Leads to Abnormalities in Cellular Recycling Process, Study Shows

News Loss of TPP1 Linked to Brain Inflammation and Nerve Cells’ Dysfunction, Mouse Study Says

News Patients Showing Signs of LINCL Should be Tested for CLN5, CLN8 Mutations, Study Says

News Nonprofit Works to Raise Rare Disease Awareness in India, Connect with U.S. Groups

News Scientists Discover New Mutation in CLN3 Gene Associated with Juvenile Batten Disease

News Brineura Over 3 Years Seen to Delay Loss of Motor-Language Skills in CLN2 Children, Trial Data Shows

News New Therapies Being Developed for Batten Disease at ‘Unprecedented’ Pace, Review Suggests

News Alpha-Synuclein Protein May Contribute Toward Batten Disease Progression

News Brexit Could Have Real Effects for UK Rare Disease Patients, Experts Warn

News Moral Dilemmas Complicate Treatment of Rare Diseases, Says Israeli Bioethicist

News Amicus Research and Gene Therapy Center To Focus on Batten, Other Diseases

News NIH Rare Disease Day Highlights Joint Networks Advancing Array of Research

News WODC 2019 Organizers Expect 1,200 to Attend Rare Disease Conference in April

News Vienna to Host RARE2019 Meeting on Rare Diseases

News Patients, Family and Friends to Take Part in Variety of Rare Disease Day Events Worldwide

News One-time Gene Therapy RGX-181 Wins FDA Rare Pediatric Disease Designation

News ICD-10 Codes, ‘Really Important’ to Rare Disease Patients, Soon Up for Fresh Consideration

News Mouse Model for CLN6 Disease Shows Sex-specific Differences in Behavior, Lifespan, Study Finds

News FDA Revising ‘Draft Guidance’ on Developing Treatments for Rare Diseases

News Mutated CLN3 Protein May Indirectly Promote Abnormal Signaling Cascades Involved in Juvenile Batten Disease, Study Finds

News CLN3 Disease Should be Considered When Diagnosing AVM, Researchers Say

News Both Rare and Classic Juvenile Batten Types Show Changes in Skeletal Muscle Cells, Study Finds

News Amicus Therapeutics Recruiting Children with CLN3 Batten Disease for Gene Therapy Trial

News Rising Healthcare Costs Strain EU Budgets Even as New Therapies Flourish

News New Mutations Linked to Infantile and Late-Infantile Batten Disease Identified in Study of Indian Patients

News CLN8 Deficiency Disrupts Cell-Signaling Pathways, Study Reports

News Mutations in CLN8 Block the Transport of Lysosomal Enzymes, Leading to Batten Disease, Study Shows

News Regenxbio’s One-time Gene Therapy RGX-181 Wins FDA’s Orphan Drug Designation

News Abeona Acquires Rights to Develop Gene Therapies Using NAV AAV9 Gene Delivery System

News Canine Models of Batten Disease May Help to Develop New Therapeutic Approaches, Review Says

News #NORDSummit – Major Issues on Table for Rare Disease Patients in US as Midterm Elections Approach

News CLN3 Deletion Affects Genes Responsible for Production of Type of Fat in Brain, Mouse Study Finds

News Researchers Dissect Mechanisms Involved in CLN5 Release Using Amoeba Model

News Researchers ID 8 Potential Biomarkers of Disease Severity in Late Infantile Batten Disease

News Compounds Similar to Flupirtine Seem Promising for the Treatment of Batten Disease, Study Reports

News CLN3 Protein Involved in Maintaining Water Balance, Batten Disease Model Shows

News Advocacy Groups, Doctors Question Rising Prices of Rare Disease Treatments

News Researchers Explore Caspase-1 as Potential Therapeutic Target for CLN3 Disease

News Amicus Therapeutics’ Expanded Portfolio Includes Potential Gene Therapies for Batten Disease

News Exome Sequencing is a Valuable Tool for Diagnosis of Pediatric Batten Disease

News Search for Biomarkers in CLN3 Mouse Model Fails to Yield Results, Study Reports

News #NORDSummit – More Than 700 Expected to Attend Oct. 15-16 Rare Disease Summit in Washington

News Researchers Use CT Scans to Monitor Long-term Brain Alterations in Sheep Models of Batten Disease

News LivaNova Launches Study to Assess VNS Therapy in Drug-resistant Epilepsy Patients

News Regenxbio Moves Toward Human Clinical Trial for Investigational Gene Therapy RGX-181

News Batten Disease Biomarker Research Influenced by Husbandry Conditions Among Mice, Study Finds

News Nerve Cells’ Dysfunction May Contribute to Neurodegeneration in Several Types of Batten’s, Mouse Study Reveals

News FDA Accepts New Drug Application for Nayzilam Nasal Spray for Acute Treatment of Seizures

News Motor Events in Juvenile Batten Disease Like Those Seen in Brain Injuries, Study Suggests

News Healx Raises $10M to Speed Therapy Discovery for Batten, Other Rare Diseases

News Experts Create New Guidelines for Optimal CLN2 Disease Management

News Discovery of Natural CLN7 Model in Type of Monkey May Help Advance Understanding of Disease

News ‘Behind These Hands,’ a Novel-in-Verse, Illustrates Affects of Batten Disease Diagnosis

News New Insight into CLN5 Protein May Help Develop New Therapeutic Strategies, Study Reports

News Common Disease Mechanisms Found Between Type of Batten Disease and Form of Adult Dementia

News Progress in Gene and Cell Therapies Reason for Hope Among Batten Patients, Review Study Says

News Scientists Find Previously Unknown Role for Protein Linked to Juvenile Batten Disease