News HHS Secretary Alex Azar Touts White House Efforts to Cure Rare Diseases

News Mitochondrial Protein’s Loss Linked to Muscle Weakness and Poor Repair in Early Study

columns, Living, Learning, Thriving - a Column by Ralph Yaniz My Adventure in a Wheelchair

News Avidity Secures $100M Funding to Advance Treatments for Myotonic Dystrophy, DMD

columns, Hawk's-Eye View - a Column by Hawken Miller How to Overcome Being Self-conscious About Using a Power Chair

News New DMD Mouse Model Helps Monitor Real-Time Dystrophin Production, Study Reports

News Experts Barry Byrne, Jerry Mendell Lead NORD Webinar on Gene Therapy

News CureDuchenne Develops Biobank to Aid Duchenne Lab Research

News Method Comparing RNA Differences Between Gene Copies May Help Find Cause of Disease, Study Suggests

News Long-term Treatment with Puldysa Reduces Lung Function Decline in DMD Patients, Study Shows

columns, You, Me and MD - a Column by Leah Leilani Improving My Digestion with Enzyme Supplements

News NS Pharma to Provide Update on its Investigational Therapy Viltolarsen in Webinar

columns, Hawk's-Eye View - a Column by Hawken Miller Expressing an Attitude of Thankfulness This Holiday

News PPMD Revamps Duchenne Registry with New App

News Rare Disease Film Festival Highlights Patient and Researcher Unity

columns, Living, Learning, Thriving - a Column by Ralph Yaniz Let’s Stay on the Cutting Edge of Research

News FDA Puts 2nd Clinical Hold on IGNITE DMD Trial of Gene Therapy SGT-001 Due to Patient Reaction

News Clinical Trial Program Testing RG6206 in DMD Boys Has Been Discontinued

columns, You, Me and MD - a Column by Leah Leilani Attack of the Bloodthirsty Mosquitoes

News Local November 9th MDA Disease Education Events Span East, West Coast

News Experimental Cell Therapy Improves Cardiac Function in Mouse Model of DMD, Study Says

News Investigational Therapy Losmapimod Targets Root Cause of FSHD, Early Study Suggests

columns, Hawk's-Eye View - a Column by Hawken Miller Goals Can Simplify Our Complicated Lives

News BIIB100 Improves Motor Function, Slows DMD Progression in Animal Models, Study Finds

News PPMD Grants Funding for Research on Cardiac Health in People with Duchenne

columns, Living, Learning, Thriving - a Column by Ralph Yaniz Prioritizing Goals in Life with MD

News Translarna Preserves Lung Function, Muscle Strength in DMD Boys, Real-World Data Show

columns, You, Me and MD - a Column by Leah Leilani I Won’t Give Up My Positive Attitude for Others

News Vamorolone Designated Promising Innovative Medicine in UK for DMD Treatment

News Novel Gene Therapy Safely Prevents Muscle Deterioration in Animal Models of DMD, Study Reports

News Vamorolone Extension Trial Supports Muscle Gains in DMD with Fewer Side Effects, Topline Data Show

columns, You, Me and MD - a Column by Leah Leilani Adjusting to Life Without My Gallbladder

columns, Hawk's-Eye View - a Column by Hawken Miller Friendships Matter: You Don’t Have to Deal with Duchenne Alone

News CureDuchenne Developing a Biobank for DMD Research

columns, Living, Learning, Thriving - a Column by Ralph Yaniz Peeling Back the Layers of Societal Barriers for People with Disabilities

News Positive Early Results Support Continuation of Phase 2 Trial of ATL1102 for DMD

News PPMD Grants to Promote Gene Therapy for Heart Disease and Patient Outcomes Research

News Catabasis Joins Jain Foundation to Advance Edasalonexent Development for Dysferlinopathy

News Dyne Therapeutics Supporting FSHD Natural History Study Underway in US, Europe

News Gene Therapy SRP-9003 Showing ‘Very Encouraging’ Results at 9 Months in Limb Girdle MD, Sarepta Reports

columns, Hawk's-Eye View - a Column by Hawken Miller The Right Job Can Help You Find Fulfillment and Purpose

News Request to Approve Viltolarsen, Exon 53 Skipping Treatment for Duchenne MD, Before FDA

News First Baby Tested for Duchenne in PPMD’s Newborn Screening Pilot Program in NY

columns, Living, Learning, Thriving - a Column by Ralph Yaniz Opportunities Abound for Travel and Accessibility

News FDA Awards $1M to Phase 2 Study of Ifetroban in Treating Heart Disease Linked to Duchenne MD

columns, You, Me and MD - a Column by Leah Leilani Honoring Serena Lawrence: How Her Life Changed Mine

News CureDuchenne Plans Oct. 11-13 ‘Futures’ Meet at California’s Disneyland

News PPMD Announces First Adult Certified Duchenne Care Center

News NORD 2019 Rare Disease Summit Set for Oct. 21-22 in Washington, DC

News Final Tests Planned into Potential Gene Therapy for OPMD, Could Support Trial in Patients

columns, Hawk's-Eye View - a Column by Hawken Miller Redefining the Word ‘Independence’

News CureDuchenne Offers One-Stop Informative Guide on Durable Medical Equipment

News Suvodirsen Receives FDA’s Fast Track Status to Treat DMD

columns, Living, Learning, Thriving - a Column by Ralph Yaniz ‘30 Days of MD’ Brings Awareness That Lasts All Year

News Acceleron Stops Clinical Development of ACE-083 as Treatment for FSHD

News New Blood Biomarkers of DMD and Glucocorticoid Response Identified

News Microdystrophin Gene Therapy Improves with nNOS Domain, Review Study Says

columns, Living, Learning, Thriving - a Column by Ralph Yaniz National LGMD Conference Builds the MD Community

News MDA Awards $6.6M to 25 Scientists Working in DMD, Other Neuromuscular Diseases

columns, You, Me and MD - a Column by Leah Leilani My Asthma Flare Inspired Me to Buy a Vogmask

columns, Hawk's-Eye View - a Column by Hawken Miller Regular Checkups Can Help You Stay Well for as Long as Possible

News BBP-418 in Development to Treat LGMD Type 2I, with Natural History Study to Start Soon

News September is MDA National Muscular Dystrophy Awareness Month

News BioNews’ MD Forum Offers Support, Community – and a Place to Vent

News Vamorolone Improves Muscle Function in DMD Boys, Phase 2a Trial Data Show

columns, You, Me and MD - a Column by Leah Leilani My Boyfriend Is Full of Ideas to Help Fight Inaccessibility

News ‘Disorder’ Film Festival Offers Look at Siblings Touched by Rare Diseases

News Jett Foundation’s Mission: ‘Making People Care About Duchenne’

columns, Living, Learning, Thriving - a Column by Ralph Yaniz Inaccessible Accessibility: It’s Time to Get Serious

News At NY Genome Center, Legal Expert Presents Ethical Dilemmas in Gene Editing

News Adults Living with DMD, Parents and Patients, Voice ‘Cautious Optimism’ for Gene Therapy in Interviews

columns, Hawk's-Eye View - a Column by Hawken Miller The Importance of Respecting and Listening to Your Parents

News Erin Frey to Lead CureDuchenne’s DMD Advocacy Efforts

News FDA Raises Concerns About Golodirsen as Potential Exon 53 Skipping Duchenne Therapy

News Fulcrum Launches Phase 2b Trial of Losmapimod for FSHD

columns, You, Me and MD - a Column by Leah Leilani My Disability Does Not Mean I Am Broken

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

columns, Hawk's-Eye View - a Column by Hawken Miller An Open Letter to Duchenne Parents

News Sarepta’s Gene Therapy Trial Continues Despite Erroneous Event Report

News Jiffy Lube Launches 8th Annual ‘Muscle Up!’ Campaign for MDA Summer Camp

columns, Living, Learning, Thriving - a Column by Ralph Yaniz Learn to Rejuvenate to Really Thrive

News Children’s Hospital of Richmond Named Certified Duchenne Care Center by PPMD

columns, Hawk's-Eye View - a Column by Hawken Miller Don’t Let Duchenne Stop You from Having Fun

News Israel’s ‘Little Steps’ Helps Duchenne Boys Catch the Waves

News Vertex Enters Duchenne Therapy Market with $1B Acquisition of Exonics

columns, Living, Learning, Thriving - a Column by Ralph Yaniz Advocacy, MD, and Dealing with the US Healthcare System

News University of Manitoba Biomedical Students Design Wheelchair Hand Warmer for MD Patients

columns, You, Me and MD - a Column by Leah Leilani I’m Not Letting My Fear of Surgery Control My Life

columns, Hawk's-Eye View - a Column by Hawken Miller Duchenne Takes My Physical Abilities, but Not My Powers of Observation

News Physical Therapy Essential for Duchenne Boys if Given with Care, Expert Says

News Oklahoma Ranks Lowest on Programs Key to Rare Diseases on NORD’s 2019 State Report Card

News Independent Review Board Clears HOPE-2 Trial to Continue Testing CAP-1002 for DMD

News Rare Disease Groups Seek Public Support to Renew Newborn Screening Act in Senate

News November Ascent of Nepal’s Mt. Everest to Raise Money for Duchenne Research

News Invitae Offering Free Genetic Testing for Muscular Dystrophy to Those Eligible in US and Canada

columns, You, Me and MD - a Column by Leah Leilani Celebrating My Tattoo Milestone and a Blessing in Disguise

columns, Hawk's-Eye View - a Column by Hawken Miller Scheduling Time for Rest Gives Me Fuel to Pursue My Passions

News Father Inspired by Son to Spearhead Duchenne Parent Project Italy

News DMD Expert Advises Parents: ‘How to Talk to School, Family and Kids About Duchenne’

columns, Living, Learning, Thriving - a Column by Ralph Yaniz The Truth About Positive Thinking and How to Achieve It