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

News Topline Results Announced for Phase 3 Trial Testing GS010 in LHON Patients

News FDA Revising ‘Draft Guidance’ on Developing Treatments for Rare Diseases

News Certain MELAS Patients Have Higher Fear of Disease Progression, Study Shows

News Healthcare Utilization of Mitochondrial Disease Patients Much More Costly Than General Population

News Genetic Mutations Affecting Protein Production Identified in Mitochondrial Disease

News Mitochondrial Surveillance Mechanism Is Critical for Genomic Stability, Study Shows

News AOX Enzyme May Alleviate Symptoms of Mitochondrial Disease, Mouse Study Shows

News Entrada Raises $59M to Advance Program to Treat Rare, Deadly Mitochondrial Disease

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

News GS010 Improves Visual Acuity, Quality of Life in LHON Patients, Phase 3 Trial Shows

News NeuroVive Secures Funding to Advance NV354 for Potential Mitochondrial Disease Treatment

News Mitochondrial DNA from Fathers Could Lead to New Therapies, Study Suggests

News Correcting Precise RNA Alterations May Restore Protein Production in MERRF, Study Suggests

News Mount Sinai’s Icahn School of Medicine Cited for Commitment to Mitochondrial Care

News New Mutations in NDUFA6 Gene Found in Four Cases of Mitochondrial Dysfunction

News NV354, Potential Therapy for Mitochondrial Diseases, Shows Promise in Preclinical Studies

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

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

News PISD is a Novel Mitochondrial Disease Gene, Case Report Shows

News Elesclomol Can Help Restore Copper Levels in Patients with Mitochondrial Diseases, Researchers Find

News $4M Grant Will Help Advance Research into NVP015 Treatment for Genetic Mitochondrial Disease

News Investigational Therapy Jotrol Shows Potential to Treat Mitochondrial Disorders, Mouse Study Suggests

News Short-Term Treatment With KH176 Lessens Depression, Improves Mood in Mitochondria Patients, Study Shows

News Patients Groups Funding Research Project Focused on Developing New Nucleoside Therapy

News Women with Mitochondrial Disease More Prone to Pregnancy Complications, Study Shows

News SLC25A4 Gene Mutation Linked to Mild Mitochondrial Myopathy in Children, Case Study Shows

News HotSpot Therapeutics Secures $45M to Advance Drug Discovery Platform

News Cerebrospinal Fluid Biomarker NfL Can Help Monitor Nerve Cell Degeneration, Study Says

News NeuroVive Licenses BridgeBio Subsidiary to Develop LHON Therapies

News Older Moms More Likely to Pass Along Mitochondrial DNA with Mutations, Study Finds

News Stealth Raises $100M to Advance Elamipretide as Treatment for Mitochondrial Diseases

News Mutations in Mitochondrial Gene ANT1 Linked to Risk of Bipolar Disease in Mouse Study

News Mutations in MRPS2 Gene Linked to Mitochondrial Dysfunction for First Time, Case Report Shows

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

News Surveys Details Reasons Mitochondrial Disease Patients Take Part – or Don’t – in Clinical Trials

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

News Phase 1 Clinical Trial Testing Potential Mitochondrial Disease Therapy KL1333 Shows Positive Results

News Mitochondrial Disease Treatment Candidate KH176 Shows Potential, Study Finds

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 Multiple Consultations, Tests and Misdiagnoses Mark Mitochondrial Patients ‘Odyssey,’ Survey Finds

News FDA Grants NeuroVive’s KL1333 Orphan Drug Status for Treatment of Mitochondrial Diseases

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

News #AAN2018 – Mutation in USMG5 Gene Causes Leigh Syndrome, Study Shows

News Some Antioxidants Show Benefits in Models of Mitochondrial Disease, Study Reports

News NIH Agency Pioneers Collaborative Research into Rare Diseases

News Genetics Foundation to Donate Adapted Bikes to Charlotte-Area Children with Genetic Conditions During Day of Caring

News Gene Mutation Leads to Early Childhood Neurodegenerative Disorder, Researchers Find

News Abnormal Protein Interaction Impairs Mitochondrial Function, May Lead to Neurodegenerative Disorders, Study Suggests

News Mitochondrial DNA Reaction Can Lead to Mutations That Cause Disease, Study Reports

News New Cellular Pathway Helps Protect Mitochondria During Stress, Study Shows

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

News Protein That Helps Our Circadian Clocks and Mitochondria Work Together Identified in Study

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

News Scientists May Have Found New Mechanism Behind Mitochondrial Disease Development

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

News New Mutations Found in Mitochondrial DNA That Cause LHON, Study Finds

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

News Enrollment Completed for Phase 2/3 Trial of Elamipretide for Treatment of Barth Syndrome

News Phase 2 Trial Finds Possible Benefits of Omaveloxolone to Mitochondrial Function at Set Dose

News Potential Gene Therapy May Improve Survival in MNGIE Patients, Mouse Study Suggests

News New Method Improves Delivery of Healthy Mitochondria To Cells Where It Is Dysfunctional, Study Shows

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

News Gene Therapy GS010 Is Safe, Well Tolerated by LHON Patients in Phase 1/2 Trial

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

News Clinical Trials to Study ChromaDex Therapy’s Effect on Mitochondrial Disease and a Heart Condition

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

News Manganese Deficiency Underlies Mitochondria Defects in Leigh Syndrome, Other Diseases, Study Finds

News Heart, Brain Very Sensitive to Mitochondrial Defects in Early Development, Study Shows

News Researchers Identify New Gene Variant Related to Childhood Mitochondrial Disease

News Distinct and New Mitochondrial Disease Seen in Children with Certain PARS2 Mutations in Study

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

News Blocking Accumulation of Stress Protein in Mitochondria May Halt PAH Progression, Study Suggests

News Neurovive’s Investigative Compound NV556 Shows Promise to Treat Mitochondrial Myopathy in Preclinical Study

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

News Compound Mimicking Coenzyme Q10 May Help Protect Neuronal Mitochondria, Study Suggests

News Study Reveals Direct Contact Between Mitochondria and Lysosome Within the Cell

News Mitochondrial Replacement Therapy Shows Promise at Preventing Disease, But Challenges Remain

News Enzyme EXD2 Is Important in Mitochondrial Protein Production, Researchers Find

News Oscillatory Vibration Training Known as SAVT Can Improve Muscle Power in Mitochondrial Disease Patients, Study Shows

News Study Shows How DNA Mutations in Healthy Mothers May Lead to Severe Mitochondrial Disease in Children

News Nutritional Supplements for Mitochondrial Diseases Need Scientific Validation, Experts Contend

News Parents of Mito Children Often Face False Medical Abuse Charges, Patient Advocates Claim

News Mitochondrial Groups Launch Pilot Phase of New Patient Care Network

News Alpers Syndrome May Be Caused by Loss of Mitochondrial DNA in Brain Cells Called Astrocytes, Study Finds

News Researchers Find Key Quality Control Mechanism in Mitochondrial Protein Production

News Genetic Background Influences Number of Mitochondrial Disease-Causing Mutations in Groups of People, Study Says

News Enteral Tube Feeding Improves Nutrition in Kids with Mitochondrial Disease, Review Suggests

News NeuroVive’s KL1333 Shows Promising Progress in Phase 1 Clinical Trial

News Mitochondria DNA Seen to Be Complex, Vary in Speed of Mutations Between People and Mice in Study

News Researchers Identify Protein in Worm that Helps Mitochondria Adapt to Stress

News Ambry Genetics-Led Research Collaboration Clears Path to Understanding Optic Atrophy in Mitochondrial Disease

News GenSight Biologics Reports GS010 Has Promise for Improving Long-term Vision in LHON Patients

News NeuroVive Selects Compound from NVP015 Program for Preclinical Studies in Mitochondrial Disorders

News FDA Grants Stealth Fast Track Designation for Elamipretide in Barth Syndrome

News Preliminary Results of Phase 2 Trial Show Khondrion’s KH176 Safe for Treating Mitochondrial Disease

News Low Coenzyme Q Is Common Among Genetic Models of Mitochondrial Dysfunction, Study Finds

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

News Patent Likely to Be Issued to Mitochon for Mitochondria-targeted Therapies