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

News Fruit Fly Research Examines Two Genes Involved in Batten Disease

News CellCept Safe, But Ineffective, in Phase 2 Study for CLN3 Disease

News Newborn Screening Test Shows Promise for Detecting Late Infantile Batten Disease, Study Shows

News Researchers Identify 5 New Mutations Linked to Form of Batten Disease

News New Mouse Model of Late Infantile Batten Disease May Help Assess Potential Therapies

News First Batten Disease Awareness Day a Milestone to Unite Global Community, Increase Knowledge

Just Published, News Neuron-Like Cells Derived from Patients May Offer Way to Better Understand and Treat Disease, Study Reports

News Study Provides More Insight into Early Signs, Progression of CLN5 Disease

News US ‘Right to Try’ Law Meets with Mix of Praise and Criticism, Including Among Those with Rare Diseases

News #ERDC2018 – When Treating Rare Disease Patients, Don’t Overlook Quality of Life, Panel Urges

News Clinical Evolution of Batten Disease in Young Adult Siblings Described in Case Report

News Protein in Specific Cells Involved in Batten Disease Neurodegeneration, Mouse Study Suggests

News New Mutation Found in Newborn with Congenital Neuronal Ceroid Lipofuscinosis, Case Study Reports

News ‘Rare Impact Awards’ Dinner Marks Orphan Drug Act and NORD at 35

News #ECRD2018 – EU Must Do More for Rare Disease Patients, Eurordis Leaders Say

News Lack of Progranulin Protein Sheds Light on Molecular Features of Batten Disease, Study Shows

News Study Examines Promising Advances in Gene Therapies to Treat Batten Disease

News More Reliable Tools Needed to Research CLN3 Protein in Batten Disease, Study Shows

News Brineura Helps Children with CLN2 Retain Motor, Language Skills, Trial Data Show

News #ECRD2018 – Patient Access and Collaboration Focus of Eurordis Meeting May 10-12

News ABO-202 Earns EMA’s Orphan Drug Status for Infantile Batten Disease

News Several Compounds Found to Correct Brain Defects in Mouse Model of CLN3 Disease

News Hair-like Structures on Cells Play Role in Batten Disease Development, Mouse Model Shows

News Neural Stem Cells from Batten Patients Can Be Used to Test New Therapies, Researchers Say

News Genome Sequencing Accurately, Rapidly Diagnosed CLN8 Variants in Boy, 8

News NIH Agency Pioneers Collaborative Research into Rare Diseases

News Gene Therapy Prevents Vision Loss Caused by CLN6 Disease in Mouse Study

News Loss of CLN7 Protein Contributes to Development, Progression of Batten Disease, Mouse Study Finds

News Key Beyond Batten Disease Foundation Fund-raiser Brings in $70,000 in February

News Inaugural Issue of the CRISPR Journal Features Study About Batten Disease

News FDA Grants Rare Pediatric Disease Designation to ABO-202 for Infantile Batten Disease

News Genome Sequencing and Its Clinical Potential Focus of NYC Rare Disease Day Event

News New Mouse Model and Gene-Editing Tool May Help Advance CLN2 Research

News ‘A Disease May Be Rare, Hope Should Never Be,’ Says Boy with SMARD at Connecticut Rare Disease Day Event

News Rare Disease Groups Welcome FDA’s Embrace of ‘Real World’ Data in Clinical Trials

News BioMarin Launches ‘RARE Scholars’ Scholarship Program for Students with Batten, Other Rare Diseases

News Brineura Approval Invigorates Research, Awareness About Other Batten Treatments

News US Lawmakers Urge Bipartisan Support for Rare Disease Research, Patients’ Needs

News Clinical Trials, Though Experimental, Often Are Best Option for Batten Patients

News Batten Disease Families Start, Rely on Foundations to Advocate, Advance Therapies

News Retrophin, Horizon Donate $3M Each to Rare Disease Institute at Children’s National

News BioNews to Cover 3 Rare Disease Day Events, Including NIH Conference

News Two Foundations Offering Batten Disease Stem Cell Lines to Researchers

News Metallothioneins Show Promise as Therapeutic Targets in Mouse Model of Batten Disease

News Patient Advocacy Groups Worldwide Plan Events to Mark Rare Disease Day, Feb. 28

News CLN2 Therapy Brineura Gets WORLDSymposium 2018 New Treatment Award

News Recommendation Against U.K. Coverage of Brineura Jolts Batten Disease Community

News FDA Names ABO-202, Potential Gene Therapy for Infantile Batten Disease, an Orphan Drug

News Rare Disease Groups, Patients Differ on ‘Right to Try’ Bill Before US Congress

News Abnormal Autophagy Is Common in Lysosomal Storage Diseases, Including Batten

News Early Detection of Retinal Dysfunction May Help Diagnosis

News NORD Fundraiser Sets 7,000-Mile ‘Bike, Run or Walk’ Goal for 7,000 Rare Diseases

News Bioinformatic Tool Finds 71% of Batten-causing Mutations in Key Protein Regions

News Severe Neurodegeneration in Newborn May Be Rare Type of Batten, Case Study Shows

News FDA Grants PLX-100 Orphan Drug Designation to Treat Batten Disease

News Mouse Model Helps Researchers Identify Processes Underlying Batten Disease Symptoms

News Review Suggests Abnormal Manganese Processing May Impact Batten Disease Progression

News Educational Intervention Needed Early in Batten Patients, Report Suggests

News Rare Disease Patient Groups Unite to Preserve Orphan Drug Tax Credit in US

News Loss of Synaptic Brain Signaling May Cause Batten Disease, Mouse Study Suggests

News Batten Organization’s Director Says Scientists Zeroing In on Treatments

News 12-year-old Spearheading $1 Million Research Fund-raiser for Sister with Batten Disease

News Children’s National, NORD Partner to Create Rare Disease ‘Centers of Excellence’

News Brain Cell Function Impaired in Early Forms of Batten Disease, Mouse Study Shows

News #NORDsummit – Alternatives to Large Placebo Trials, Grant Awards Among Ways FDA Supporting Rare Diseases, Chief Says

News Brineura, for Batten CLN2, Named One of Year’s Top Health Innovations by Popular Science

News #NORDsummit – Despite Criticism, Orphan Drug Act Is Working to Advance Needed Treatments, FDA Says

News Gene Therapy Decreased Protein Aggregates in Animal Model of Batten Disease, Study Finds

News Collection of Batten Disease Stem Cell Lines Available for Research, Organizations Announce

News Utah Family Loses 3 Children in 3 Consecutive Days to Batten Disease

News Yeast Models of Batten Disease May Speed Up Treatment Discovery, Researchers Argue

News U.K. Batten Group Launches ‘BATCure’ Survey for Patients and Families Across Europe

News Photosensitivity May Indicate CLN Type 2 Disease in Youngsters, Study Suggests

News Hurricane Harvey Leads to Two Young Friends with Batten Disease Reuniting at Memphis Hospital

News Potential Batten Biomarkers Identified Through Protein Analyses

News Abeona Will Give Update on Its Gene Therapy Programs in R&D Day Set for October

News Batten Disease Clinical Trials Enrolling Patients to Evaluate Gene Therapies

News Polaryx’s Treatment Candidate for LINCL Granted FDA Orphan Drug Status

News Charlotte and Gwenyth Gray Foundation Funds Clinical Trial for Rare Batten Disease Variant

News National Organization for Rare Disorders Gives Batten Disease Community a Voice in Washington

News Current Batten Therapeutics Targeting Brain Not Enough to Stop Disease in Other Organs, Canine Study Suggests

News CLN5 Protein Loss in Batten Disease Led to Fewer Functional Neurons in Mouse Study

News North Carolina’s Indo Jax Surf Charities Helps Kids With Batten Disease, Other Special Needs

News Infantile Batten Disease May Begin in Spinal Cord Rather Than Brain, Study Suggests

News Young Batten’s Patient Inspires Lawmakers to Create Rare Disease Advisory Council

News EU and US Regulators Offer Recommendations on Developing Therapies for Children’s Diseases

News FDA Marks Potential Gene Therapy for Juvenile Batten Disease as Orphan Drug to Speed Development

News ‘Love, Kennedy’ Film Chronicles Life of ‘Amazing’ Teen with Batten Disease

News Induced Pluripotent Stem Cell Models Aid in Study of Lysosomal Storage Disorders, Including Batten Disease

News Cholesterol Therapy Gemfibrozil Improves Disability and Lifespan of Mice with Batten Disease, Study Reports

News Brineura Approved as Batten Treatment in Both Europe and US as Clinical Studies Continue