News PPMD Awards $100K for Immune Cell Profiling Research for Duchenne and Becker at UCLA

columns, You, Me and MD - a Column by Leah Leilani Choosing the Right Dog Breed for My MD Lifestyle

columns, Hawk's-Eye View - a Column by Hawken Miller Traveling the World Is Possible, Even with Duchenne

News Sarepta Acquires Rights to Novel Gene Therapy Candidate to Treat Limb-Girdle MD

columns, Living, Learning, Thriving - a Column by Ralph Yaniz Let’s Prepare Ourselves for the Trials of a Lifetime

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

News Duchenne Teen Hits the Runway at Las Vegas Fashion Show

columns, You, Me and MD - a Column by Leah Leilani My Search for a Comfortable Bra as a Woman in a Wheelchair

columns, Hawk's-Eye View - a Column by Hawken Miller Video Games Let Me Be Competitive Without the Need to Be Physical

News #AANAM – FSHD Patients ‘Very Enthusiastic’ About Taking Part in Clinical Trials, Research Suggests

News PPMD Launches Pilot Duchenne Newborn Screening Program in NY

News #AANAM — New TTN Gene Variants Potentially Linked to Limb-girdle MD, Study Says

columns, Living, Learning, Thriving - a Column by Ralph Yaniz Find Your Communities and Gain a Sense of Belonging

News #AANAM — Very Young Boys with DMD May Have Measurable Early Signs of Motor Skill Delay, Study Suggests

News #AANAM — Phase 1b/2 Trial Of RG6206 in Boys With DMD Supports Safety, Positive Muscle Effects

News Gene Therapy Candidate GALGT2 Found Safe in Parent Project-Funded Toxicology Study

columns, Hawk's-Eye View - a Column by Hawken Miller Dealing with Duchenne and the Power of a Good Night’s Sleep

News EGF Protein Preserves Muscle Strength, Promotes Muscle Regeneration in DMD Mice, Study Shows

columns, Living, Learning, Thriving - a Column by Ralph Yaniz I Live Creatively to Power Past Obstacles

News PALS Uplifts the Spirits of Rare-Disease Patients by Offering Free Flights

News Lab Test Allows Prenatal Diagnosis of One Type of Congenital MD, Study Finds

columns, You, Me and MD - a Column by Leah Leilani Grieving the Loss of My Furry Friend

columns, Hawk's-Eye View - a Column by Hawken Miller Achieving Balance in College Was Key to My Success

News Repairing Damage to Muscles May Be Matter of Getting Proteins to Trigger Stem Cells, Early Study Finds

News Fulcrum Takes Charge of Compound That Might Treat Root Cause of FSHD, Plans Phase 2 Study

News Wave’s Exon 51-Skipping Therapy Safe, Well-Tolerated in DMD Boys, Phase 1 Trial Results Show

columns, Hawk's-Eye View - a Column by Hawken Miller Finding My Purpose in Life Despite Duchenne Muscular Dystrophy

News PPMD Wins Bipartisan Legislative Backing of Its Muscular Dystrophy Goals

columns, Living, Learning, Thriving - a Column by Ralph Yaniz True Accessibility for All: No Roadblocks, Please

News Pamrevlumab, Potential Treatment for Fibrosis in DMD Patients, Named Orphan Drug by FDA

columns, Hawk's-Eye View - a Column by Hawken Miller Learning to Be Social with Duchenne

News Audentes Therapeutics Expanding Treatment Candidates for Duchenne MD and Myotonic Dystrophy Type 1

Duchenne Muscular Dystrophy News, News Single Dose of CRISPR Gene Therapy May Succeed as Long-term Treatment for DMD, Mouse Study Shows

columns, Living, Learning, Thriving - a Column by Ralph Yaniz I Push My Limitations by Setting Goals

News Raising Both Dystrophin and Utrophin Levels May Rescue Muscles in DMD, Mouse Study Suggests

columns, Hawk's-Eye View - a Column by Hawken Miller Focus on What You Can Do Rather Than What You Can’t

News RNA-targeting Compound Shows Ability to Limit Muscle Damage in Early Myotonic Dystrophy Type 1 Study

News Casimersen Shows Promising Phase 3 Results for DMD, May Open Door for FDA New Drug Application

columns, Living, Learning, Thriving - a Column by Ralph Yaniz ‘Living, Learning, Thriving’: The Start of Something New

News DMD Gene Therapy Showing ‘Very Encouraging’ Results at 9 Months in Phase 1/2 Study, Sarepta Reports

columns, You, Me and MD - a Column by Leah Leilani Looking at the Complexities of Mitochondrial Myopathy and Autism

columns, Hawk's-Eye View - a Column by Hawken Miller The Art of Asking for Help

News Duchenne Family Assistance Program Expands to Include Resource-Finder Tool

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

News Santhera Filing for EMA Conditional Marketing Authorization for Idebenone to Treat Respiratory Issues in DMD

News PPMD, Cure SMA Join Prometheus in Data Collection Partnership

columns, Hawk's-Eye View - a Column by Hawken Miller Introducing ‘Hawk’s-Eye View,’ a Duchenne Column

News Boys with Duchenne Muscular Dystrophy at High Risk for Bone Fractures

News CRISPR-Cas9 Corrects Common DMD-causing Mutation in Mice and Human Cells

News New Enhanced Microdystrophin Versions May Help Restore Muscle Function in DMD, Mouse Study Suggests

columns, You, Me and MD - a Column by Leah Leilani My Birth Control Injections Have Restored My Quality of Life

News Firefighters to Hike 130 Miles to Raise Awareness of Neuromuscular Diseases and Promote Newborn Screening

News Vamorolone Safely Aids Skeletal and Heart Muscle, Animal Study Finds

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 MDA’s 37th #MDAShamrocks Fundraiser is Underway

columns, You, Me and MD - a Column by Leah Leilani My First Time Attending the LA Abilities Expo

News CureDuchenne Sets Calendar for Workshops, Sessions and ‘Futures’ Conference

News MYO-101 Enables Production of Missing Protein in Children with LGMD2E, Trial Shows

News Raxone (Idebenone) Shows Promise as DMD Therapy in SYROS Trial

News NIH Rare Disease Day Highlights Joint Networks Advancing Array of Research

columns, You, Me and MD - a Column by Leah Leilani Join Us in the New Muscular Dystrophy News Forums

News Centogene Collaborates with Sarepta to Identify DMD Patients in the Middle East and North Africa

News Edasalonexent May Limit Disease Progression in Boys with Duchenne MD, Phase 1/2 Trial Shows

columns, You, Me and MD - a Column by Leah Leilani What Teachers Should Know About Students with Disabilities

News New Device Enables Home Monitoring of Respiratory Function in Teens with DMD, Study Reports

News WODC 2019 Organizers Expect 1,200 to Attend Rare Disease Conference in April

News Vienna to Host RARE2019 Meeting on Rare Diseases

News Edasalonexent Leads to Normal Growth, Other Benefits in Boys with DMD, Phase 1/2 Trial Shows

News MDA Releases Duchenne Fact Sheet, Survey Results Pointing to Need for Knowledge of Clinical Trials and Genetic Tests

News Gene Therapy SGT-001 Shows Signs of Microdystrophin Production in Muscles of Boys in IGNITE DMD Trial

News Golodirsen, Potential Exon 53 Skipping Therapy for Duchenne, Under Priority Review by FDA

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

News Parent Project Muscular Dystrophy Takes Its ‘End Duchenne Tour’ on Road Again for 2019

columns, You, Me and MD - a Column by Leah Leilani Tips to Alleviate Symptoms of Constipation

News Duchenne Patients Again Being Treated in HOPE-2 Trial of Potential Cell Therapy, Capricor Announces

News Defying the Odds of Living with Duchenne, Decade After Decade

News PPMD Funds Gene Therapy Research Aimed at Improving Cardiac Function in DMD Patients

columns, You, Me and MD - a Column by Leah Leilani I Can’t Do Everything, and That’s OK

News ICD-10 Codes, ‘Really Important’ to Rare Disease Patients, Soon Up for Fresh Consideration

News DMD Increases Susceptibility to Rare, Aggressive Muscle Cancer, Mouse Study Suggests

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

News Popeyes Group Launches 15th Annual Campaign to Help MDA

News ICER Document Reviews Effectiveness of 3 Duchenne MD Therapies

columns, You, Me and MD - a Column by Leah Leilani Finding the Perfect Wheelchair Blanket

News Phase 2b Trial Testing Vamorolone Now Enrolling Boys Ages 4-6 with DMD

News Cell Therapy CAP-1002 Leads to Heart, Skeletal Muscle Improvements in DMD Patients, Phase 1/2 Trial Shows

News Connecticut Man With Limb-girdle MD Inspires Others

News PPMD Publishes 10-Year Duchenne and Becker Patient Registry Report

columns, You, Me and MD - a Column by Leah Leilani Seeing Both Sides of ‘The Upside’

News New Tool to Help With Virtual Research and Care Management for Duchenne Patients

News Trial Testing WVE-210201 in DMD Selected for FDA Pilot Program

News Sarepta and Aldevron Partnering on Gene Therapy Program for DMD and LGMD

columns, You, Me and MD - a Column by Leah Leilani I’ve Learned How to Deal with Judgment About My Disease

News DMD Hub Works to Connect Patients with Clinical Trials, Promote Study Sites Across UK

News PPMD Awards $329K to Researchers Looking to Better Understand Promising Duchenne Therapies

News Phase 3 Trial of Edasalonexent in DMD Now Enrolling Across US, Global Sites to Follow

News Capricor Places Hold on Phase 2 Trial of Cell Therapy After Patient Has Allergic Reaction

News Myonexus’ MYO-102 Earns FDA’s Orphan Drug Status for LGMD 2D

News Project Hercules Enlists Industry’s Help to Make Duchenne Therapies Affordable