News #PPMD2018 — ‘Mitchell’s Journey’ Supports Duchenne Research, Helps Families Cope with Loss

News Phase 3 Trial of Oral Edasalonexent as Potential DMD Treatment to Open Soon, Catabasis Announces

News #PPMD2018 – Nebraska Mother of 3 Duchenne Boys: ‘We’ve Made a Choice to Be Positive’

News #PPMD2018 – Don’t Overlook Dental and Digestive Issues, Duchenne Experts Advise

News Potential DMD Therapy Now in Phase 1 Trial, WVE-210201, Likely To Be Named Orphan Drug in EU

News #PPMD2018 – Duchenne Research ‘Has Come a Long Way,’ PPMD Says at Meeting

News 4 Approaches to Treating Duchenne Muscular Dystrophy Highlighted at PPMD Conference

columns, You, Me and MD - a Column by Leah Leilani Managing Body Temperature with Heat Intolerance

News Cognitive Behavioral Therapy May Help Patients with Myotonic Dystrophy Type 1, Trial Shows

News Summit Therapeutics Ends Development of Ezutromid Therapy for DMD After Trial Failure

News UK Agency Renews Early Access Program for Raxone to Treat Duchenne MD

columns, You, Me and MD - a Column by Leah Leilani Interacting with Kids Who’ve Never Been Around a Wheelchair

News Way to Accurately Measure Changes in Ankle Muscles in Myotonic Dystrophy Type 1 May Aid Clinical Trials

News Microdystrophin Gene Therapy Shows Promising Interim Results in Phase 1/2 Trial

News Clinical Hold on Duchenne Gene Therapy Trial Lifts, IGNITE DMD Again Enrolling Patients

columns, You, Me and MD - a Column by Leah Leilani Planning My Summer with Both Fun and Reality in Mind

News Rehab Is Still Major Goal in New, Updated CDC Guidelines for Duchenne Management

News Proteins That Regulate Gene with Critical Role in FSHD Development Identified in Study

News Hockey Player Hosts 8th Annual Golfing Event to Raise Money for CureDuchenne

columns, You, Me and MD - a Column by Leah Leilani Dating with a Disability: Part 2

News FDA Names Potential FSHD Therapy GBC0905 an Orphan Drug to Speed Its Development

News CHMP Opposes Approval of Exondys 51 to Treat DMD in Europe

News Canada Trails US in Patient Access to Rare Disease Therapies, CORD President Says

News Duchenne Trials Aren’t Easy on Families or Patients, Even for Those Able to Qualify, PPMD Founder Says

columns, You, Me and MD - a Column by Leah Leilani Dating with a Disability: Part 1

News EU’s CHMP Recommends Translarna to Treat Younger DMD Children After FDA Rejection

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

News #ECRD2018 – Duchenne Needs ‘Thinking Outside the Box’ to Advance Treatments But Investors Taking Note, Advocate Says

News #ECRD2018 – Genome Editing Might Be ‘Cure’ for Rare Diseases But Ethical Guidelines Needed, Panel Says

News Artificial Muscles May Lead to Personalized Treatments for MD, Study Suggests

columns, You, Me and MD - a Column by Leah Leilani Learning to Embrace Relapse with Muscular Dystrophy

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

News Early Study of Exon 45 Skipping Therapy, DS-5141, in DMD Patients Shows Safety But Mixed Effectiveness

News ##ERDC2018 – New EU Law to Protect Patient Data Could Affect Rare Disease Registries

News Sarepta and Invitae Join to Speed Diagnosis of Duchenne Patients, Advance Trials of Potential Therapies

columns, You, Me and MD - a Column by Leah Leilani My Journey to Love My Chronically Ill Body

News FDA Grants Rare Pediatric Disease Status to Myonexus’ MYO-101 for LGMD 2E

News Myoda (Sarconeos) Earns FDA’s Orphan Drug Designation for Duchenne MD

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 Sarepta, Myonexus Collaborating to Advance Gene Therapies for LGMD

columns, You, Me and MD - a Column by Leah Leilani 12 Things to Pack for Your Hospital Stay

News New Exon 51 Skipping Therapy in Phase 1 Safety Trial Explained by Sarepta CEO in Webinar

News Approved Anti-inflammatory Seen to Increase Muscle Strength and Function in DMD Mice in Study

News PPMD Marks 10th Year of Duchenne Registry, Largest Repository of DMD Patient Data

News Capricor Recruiting Duchenne Patients for Phase 2 Trial of CAP-1002

News Sarepta Therapeutics Launches ‘Route 79, the Duchenne Scholarship Program’

News Acceleron’s ACE-083 Therapy Candidate for FSHD Earns FDA’s Fast Track Designation

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

columns, You, Me and MD - a Column by Leah Leilani Marriage While Raising a Disabled Child: An Interview with My Parents

News #AAN2018 – 1 Year of Edasalonexent Use Significantly Slows DMD Progression, MoveDMD Trial Reports

News Gene Therapy MYO-101 Receives FDA’s Orphan Drug Status for LGMD2E Treatment

News People with Disabilities Like MD Actively Seek Work, Overcome Barriers, Survey Finds

News Human Genome Project Team at NIH Honored for Advancing Research into Genetic Diseases

News #AAN2018 – Early Data Supports Golodirsen in DMD Patients Amenable to Exon 53 Skipping

News #AAN2018 – Clinical Trials Re-Analysis Shows Emflaza Potential to Preserve Motor Function in Boys with DMD

News #AAN2018 — Exondys 51 Found to Be Safe in Young Infant with DMD, Preliminary Data Shows

columns, You, Me and MD - a Column by Leah Leilani Noticing the Blessings in Everyday Life

News UT Researcher, with Cure Duchenne Support, Launches Company to Treat DMD Using CRISPR/Cas9 Technology

News NIH Agency Pioneers Collaborative Research into Rare Diseases

News Pfizer Launches Phase 1b Clinical Trial for Mini-Dystrophin Gene Therapy to Treat DMD

columns, You, Me and MD - a Column by Leah Leilani Breaking the Stigma of Asking for Help

News Genetic Compensation Mechanism Could Aid in Treatment of Muscle Disorders, Study Suggests

News PPMD Names PerkinElmer to Conduct Genetic Tests for Its Decode Duchenne Program

News Santhera’s Omigapil Found Safe and Well-Tolerated in Young CMD Patients in Phase 1 Trial

News Connecticut Neurologist Upbeat on New Therapies for DMD, Other Disorders

News New Gene Editing Strategy Leads to Much Longer Protein Production

columns, You, Me and MD - a Column by Leah Leilani Making My Bathroom Wheelchair-accessible

News Tamoxifen and Evista Improved Muscle, Other MD Functions in Mouse Study

News Tiny Spear-like Nanotubes May Enhance Delivery of Gene Therapies, Study Suggests

columns, You, Me and MD - a Column by Leah Leilani Fitbit Is Not Just for the Able-bodied

News Parent Project Muscular Dystrophy Hosting Annual DMD Conference in Arizona

News New CDC Guidelines Address Emerging Needs of Duchenne Care

News AMO Pharma’s Myotonic Dystrophy Therapy Improves Cognition and Function, Phase 2 Trial Shows

News Expert Uses Webinar to Explain the Science Behind Gene Therapies for Duchenne MD

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

News DMD Trial of Dystrophin Gene Therapy SGT-001 Put on Clinical Hold by FDA

News Sarepta to Seek Accelerated US Approval of Duchenne MD Therapy Golodirsen

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

columns, You, Me and MD - a Column by Leah Leilani Finding the Balance Between Style and Comfort

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

News Vietnamese Wins Kovalevskaya Award for Prenatal Diagnosis of Duchenne MD

News Summit Therapeutics Expands PhaseOut DMD Clinical Trial of Ezutromid

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

News Plasticity Becomes Authorized Care Provider for Children with MD in Special Florida Program

columns, You, Me and MD - a Column by Leah Leilani What I Use to Prevent Neck Pain

News ATL1102, Investigational Therapy for Duchenne Muscular Dystrophy, Approved for Phase 2 Trial

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

News Ezutromid Significantly Reduces Muscle Inflammation, Interim Analysis of PhaseOut DMD Trial Shows

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

columns, You, Me and MD - a Column by Leah Leilani How to Buy a Wheelchair-accessible Van

News Stem Cell Therapy Improved Duchenne MD Patients’ Heart Disease, Study Shows

News Duchenne MD Patients with Respiratory Decline Gain Access to Raxone in US

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

News Phase 3 Trial of Translarna’s Long-term Efficacy Enrolling Duchenne MD Patients with Nonsense Mutations

columns, You, Me and MD - a Column by Leah Leilani Growing Thicker Skin

News New Device Measures Cell Strength Faster, More Accurately, Aiding in Search for MD Therapies

News 4th STRIVE Awards Program Opens to Groups Promoting DMD Advocacy and Care, PTC Announces

News Catabasis Announces Global Phase 3 Trial of Edasalonexent for DMD Boys

News New CRISPR Strategy Corrects Wider Range of Mutations Responsible for DMD