News mTOR Inhibitors May Benefit Some Children With Mitochondrial Diseases, Case Series Suggests

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

News Protein’s Inability to Bind, Stabilize Mitochondrial Membrane Drives Optic Atrophy, Study Suggests

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 Trial of Cholesterol Medication in Barth Syndrome Patients Opens in UK

News European Initiative Targets Diagnosis, Treatment of Rare Diseases

News GenSight Completes Enrollment Early for Phase 3 Trial of GS010 Gene Therapy

News New Lab Model Could Shed Light on Mitochondrial Epilepsy and Therapies

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

News Phase 1/2 Trial of Mitochondrial Cell Therapy for Pearson Syndrome Starts Dosing

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

News Eurordis Unveils Integrated-care Initiative for Rare Disease Patients

News Increasing mtDNA Levels Holds Potential for Tackling Mitochondrial Diseases, Mouse Study Suggests

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

News Symptoms Vary Greatly in People with MT-ATP6 Mutations and Better Tests Needed, Study Says

News Kidney Disorder Therapy May Be Effective in Mitochondrial Disease, Study Suggests

News Reneo Pharmaceuticals Raises $50M to Support Development of Therapies for Mitochondrial Diseases

News Top-line Results Expected This Year for Phase 3 Study Testing Elamipretide Injections for Mitochondrial Myopathy

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

News GS010 Gene Therapy Continues to Improve Vision Clarity in LHON Patients, New Phase 3 Data Show

News Clinicians Should Consider Different Wording in Cases of Unconfirmed Mitochondrial Disease, Study Suggests

News MDA Conference Bringing Neuromuscular Disease Experts to Orlando to Share ‘New Era’ in Treatment, Research

News New Phase 1 Trial of Potential Treatment for Mitochondrial Diseases, KL1333, Enrolling in UK

News University of Arkansas Team Awarded NIH Grant to Find Better Diagnostic Tools for Mitochondrial Diseases

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 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