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

News As PPMD Turns 25, Founder Pat Furlong Reflects on Advances in Duchenne

columns, You, Me and MD - a Column by Leah Leilani Experiencing the Healing Effects of Medical Marijuana

columns, Hawk's-Eye View - a Column by Hawken Miller Meaningful Connections Help Me to See the Possibilities

News MDA Awards $389K to UCLA Scientist to Develop Potential DMD Treatment Targeting Sarcospan

News European Initiative Targets Diagnosis, Treatment of Rare Diseases

News Cell Therapy CAP-1002 Improves Muscle, Lung Function in Boys with DMD, Interim Data Show

columns, Living, Learning, Thriving - a Column by Ralph Yaniz ‘Disabled’ vs. ‘Having a Disability’: Debating the Power of Words

News Pfizer’s Gene Therapy Holds Promise in Treatment of DMD, Early Data Show

columns, Hawk's-Eye View - a Column by Hawken Miller UberWAV Is the Next Best Thing for Accessible Transportation

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