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

News Retina Thickness Linked to Neurodegeneration in Spinal Cord of ALD Patients, Study Finds

News Three New Mutations Associated With ALD Identified in Iranian Study

News Diffusion MRI Shows Potential as Sensitive Measure of ALD Progression, Study Shows

News Rare Disease Film Festival Highlights Patient and Researcher Unity

News Cell-based Gene Therapy Represents ‘Hope’ for X-ALD Patients, Review Study Suggests

News Aging Raises Risk of Women Developing ALD Symptoms or More Severe Disease, Study Suggests

News Researchers Develop Efficient Method to Screen for ALD in India, Study Reports

News Immune Cells in Blood Samples from X-ALD Patients May Predict Response to Treatment, Study Says

News Most ALD Patients Without MRI Lesions Retain Normal Cognitive Function, Study Suggests

News Researchers Discover New Genetic Mutation Among 6 Chinese Men with AMN

News Symptoms of Peroxisomal Disorders Like X-ALD Often Mistaken for Another Disease, Study Finds

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

News Magenta Therapeutics’ Stem Cell Therapy MGTA-456 Earns FDA’s Regenerative Medicine Advanced Therapy Designation

News Clinical Trial Data Support Safety, Efficacy of Lenti-D for Boys with CALD

News Lack of SIRT1 Likely Contributes to Neuroinflammation in X-ALD, Study Says

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 Off-Label MS Therapy Rituximab Fails to Stop Progression of Advanced Childhood Cerebral ALD, Case Report Finds

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

News Blood Stem Cell Transplant Feasible, but Better Before Onset of Neurological Complications, Case Report Shows

News First Clinical Trial of VK0214 to Treat X-linked ALD Planned for Early 2020, Viking Says

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

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

News Report Describes New ABCD1 Variant Linked to X-ALD in Chinese Boy

News Autism and ADHD Possible Early Signs of ALD, According to Case Report

News European Initiative Targets Diagnosis, Treatment of Rare Diseases

News Hyperpigmentation Should Be Considered in Diagnosing X-ALD, Case Report Says

News Report of Siblings with X-ALD Highlights Different Disease Manifestations

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

News Screening, Early Monitoring of Adrenal Glands Help Identify ALD in Newborns, Report Says

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

News APOE4 Variant Linked to Poorer Neurological Outcomes in Boys with Cerebral ALD, Study Finds

News Eurordis Unveils Integrated-care Initiative for Rare Disease Patients

News Analysis of Newborn Screening for X-ALD in Minnesota Reveals Under-reported Prevalence

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

News Combo of Antioxidants a Potential Therapy for Adrenomyeloneuropathy, Trial Suggests

News Early X-ALD Signs, Common Family Concerns Collected in Registry Study

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

News #AANAM – MGTA-456 Cell Therapy Shows Promise in cALD Patients 6 Months After Transplant, Trial Update Shows

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

News #AANAM – High-dose Biotin Capsule Shows Promise in X-ALD

News Brain Resident Immune Cells May Play Role in X-ALD Progression, Study Suggests

News Discovery of Signaling Cascade in ALD May Open Door to Trials of Tecfidera

News Cholesterol-derived Molecule 25-HC May Hold Potential for Severe Type of X-ALD

News Study Describes Two Atypical Cases of Adult-onset ALD

News 20-Year Survival Seen in 73% of ALD and Other Metabolic Disorder Patients After Transplant, Study Reports

News Blood Stem Cell Therapy MGTA-456 Showing Signs of Efficacy in Cerebral ALD, Early Trial Data Show

News Blood Stem Cell Transplant May Slow Cognitive Decline in Adult Cerebral ALD

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 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 More Sensitive Tools Needed to Assess ALD Spinal Cord Disease Progression in Women, Study Contends

News Benefits of X-ALD Newborn Screening in U.K. Outweigh Costs Incurred, Study Shows

News Brain Imaging, Tests Help Diagnose Rare Phenotype of X-linked ALD

News Risk of Adrenal Insufficiency in ALD Is Greatest from Ages 1-10, Study Says

News Abnormalities in Bile Acid Production Found in Patient with X-linked ALD, Case Study Reports

News Targeted Genetic Testing Is Critical for X-Linked ALD Diagnosis in Atypical Cases, Report Shows

News Leukodystrophy Diagnosis Less Likely in Children of Racial, Ethnic Minorities

Just Published, News Disease Severity, Transplant Source Influence Long-term Success in Childhood Cerebral X-ALD 

News Robust Donor Cell Transplant Required for Blood-brain Barrier Repair in cALD Patients, Study Says

News Changes in Cell Lipids May Help Explain Symptom Severity in X-ALD, Study Suggests

News Early Diagnosis and Stem Cell Treatment Can Stop CALD Disease Progression, Study Finds

News Abnormal Immune Response May Underlie Severe Brain Inflammation in Cerebral ALD, Study Suggests

News Data Point to Promise of Stem Cell Therapies for CALD Patients, Bluebird Bio Reports

News NeuroVia Starts Phase 1/2 Trial to Evaluate NV1205 in Childhood Cerebral Adrenoleukodystrophy

News Phase 2/3 Trial of MIN-102 as Potential Treatment for AMN Ready to Dose Patients, Minoryx Reports