News Exondys 51 Superior to Glucocorticoids in Preserving Respiratory Health of Duchenne Boys, Analysis Shows

News $500K Gift to MDA Summer Camp Will Honor Robert Bennett Legacy

columns, Living, Learning, Thriving - a Column by Ralph Yaniz The Speak Foundation Speaks Loudly: A Prelude to September in Chicago

News Macedonian Gaucher Activist Publicizes Plight of Rare Disease Patients

News Potential DMD Treatment, Pamrevlumab, Helps to Preserve Lung, Heart and Upper Limb Function in Youth, Early Trial Data Show

columns, Hawk's-Eye View - a Column by Hawken Miller Here’s How I Explain My Experience with Duchenne to Others

News MDA Awards Locana $550K to Advance Potential RNA-targeting Therapy for Myotonic Dystrophy

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

News Raising Levels of Protein, Sarcospan, Seen to Benefit Cardiac Muscles in Mouse Model of DMD

columns, Hawk's-Eye View - a Column by Hawken Miller You Can Experience the Beauty of the Outdoors, Even with Duchenne

News Enrollment Progressing Quickly in PolarisDMD Trial Testing Edasalonexent, Catabasis Says

News Edasalonexent, Potential DMD Therapy in Phase 3 Trial, Seen to Preserve Bone Health in Test in Mice Against Corticosteroid

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

News European Agency OKs Marketing Authorization for Puldysa to Treat Respiratory Decline in Duchenne MD

columns, You, Me and MD - a Column by Leah Leilani Book Review: ‘Strangers Assume My Girlfriend Is My Nurse’

columns, Hawk's-Eye View - a Column by Hawken Miller Moving into a New Life with Duchenne

News Popeyes’ Group Raises $681K for the MDA in 15th Annual Fundraising Drive

News Eurordis Unveils Integrated-care Initiative for Rare Disease Patients

News Biophytis Teams With AFM-Telethon to Advance Sarconeos as Duchenne MD Therapy

columns, Living, Learning, Thriving - a Column by Ralph Yaniz Choosing an E-bike: Some Things to Consider

News YourDNA and Cure Rare Disease Collaborate to Raise DMD Awareness, Advance Genomics

columns, You, Me and MD - a Column by Leah Leilani The Battle Between Mitochondrial Myopathy and Makeup

columns, Hawk's-Eye View - a Column by Hawken Miller Expressing Myself on YouTube with a Disability

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

News PTC Therapeutics Gets FDA Approval for Emflaza for Ages 2-5

News DMD Patients Are Living Longer, While New Cases Hold Steady, Study Finds

columns, Living, Learning, Thriving - a Column by Ralph Yaniz It’s Essential to Be Your Own Health Advocate

News Blocking Protein That Hinders Muscle Growth Possible Way of Treating LGMD1, Mouse Study Suggests

News Vertex Acquires Exonics Therapeutics, Advancing Gene-editing Treatment for DMD

columns, Hawk's-Eye View - a Column by Hawken Miller Scheduling Out Life Makes Living with a Disability Easier

News Researchers Awarded $175K Grant from Parent Project Muscular Dystrophy to Optimize CRISPR/Cas9 for DMD Gene Therapy

columns, Living, Learning, Thriving - a Column by Ralph Yaniz Uniting to Effect Positive Change

News Santhera Collaborating With Swiss Researchers to Develop New Gene Therapy for LAMA2 MD

columns, Hawk's-Eye View - a Column by Hawken Miller The Secret to Finding the Right Friends

News ‘Serious Concerns’ Raised by Watchdog Group on Cost-effectiveness of DMD Therapies

News Santhera Seeks European Approval of Puldysa to Address Respiratory Problems in DMD

News With Zolgensma’s Approval, Scientists Pursue Similar Gene Therapies in Duchenne

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

News FinancialForce Helps Boost Muscular Dystrophy Association’s Fundraising Efforts

columns, Living, Learning, Thriving - a Column by Ralph Yaniz Moving into the Future: Assistive Technology Can Make a Difference

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