News #NORDSummit – More Than 700 Expected to Attend Oct. 15-16 Rare Disease Summit in Washington

News Nonprofit Coalition to Cure Calpain 3 Funds 2 Gene Therapy Research Projects for LGMD2A

News Potential DMD Therapy Vamorolone Shows Positive Effects in Phase 2a Trial, Study Reports

columns, You, Me and MD - a Column by Leah Leilani A Girl’s Best Friend: Thoughts on Mobility Assistance Dogs

News Obsessive Compulsive Disorders Evident in Duchenne Patients and in Need of Treatment, Study Says

News Gene Editing Restored Dystrophin Levels in Dog Model of Duchenne MD, Study Reports

News STRIVE Awards Recognize Efforts of Duchenne MD Patient Groups Worldwide

columns, You, Me and MD - a Column by Leah Leilani Listening and Surrendering to My Body Isn’t Always Easy

News Numotion, Sing Me a Story Partner to Help Children with Neuromuscular Diseases Turn Life Stories to Songs

News Pfizer Stops Phase 2 Clinical Studies of Domagrozumab in DMD Boys

News PPMD and Jett Foundation Among Groups Gearing Up for Sept. 7, World Duchenne Awareness Day

columns, You, Me and MD - a Column by Leah Leilani Finding Doctors Who Understand Your Disease

News FSH Society Holding ‘Walk & Roll’ Event in Pacific Northwest to Raise Funds, Awareness for FSHD

News Service Dogs Just One Way Israel’s ‘Little Steps’ Assists Duchenne, Becker Patients

News MDA Awards $9.9M to 34 Projects Targeting Neuromuscular Diseases

News Canadian Collaboration Gives Funding Boost to Duchenne MD Research

News Long-term, Daily Glucocorticoid Use Raises Risk of Spinal Fractures in Duchenne Boys, U.K. Study Suggests

News Michigan Mom Who Lost One Son to Duchenne Works to Give Fresh Hope to the Other

News WVE-210201 Receives FDA Orphan Drug, Rare Pediatric Disease Designations for DMD

columns, You, Me and MD - a Column by Leah Leilani Seeing Both Sides of the Plastic Straw Controversy

News Jiffy Lube, MDA Join Forces for 7th Annual MUSCLE UP! Campaign

News 2 Nonprofits Join Forces to Help Duchenne Patients Fight Insurance Denials

News First Patient Enrolled in Phase 2 Trial of MNK-1411, Mallinckrodt’s Investigational Therapy for DMD

News Arizona Woman with Duchenne is a Rarity Among the Rare

columns, You, Me and MD - a Column by Leah Leilani How Lyrica Has Affected My Relationship with Food

News $700,000 MDA Grant to Help Establish LGMD Clinical Research Network

News New Online Resource, DuchenneXchange, and Its Offerings Explained in Aug. 7 Webinar

News Family’s Quest for ‘Overlooked’ Duchenne Treatments Leads to Breast Cancer Medicine Tamoxifen

News FDA Places Hold on Phase 1/2 Trial of Sarepta’s Gene Therapy for DMD

News Consortium.AI to Advance New Treatments for Duchenne Using Artificial Intelligence

News MDA Hellas Gives Greek SMA, Duchenne Patients Reason to Hope

News In Greece, One Family’s Struggle with Duchenne

columns, You, Me and MD - a Column by Leah Leilani Why I Identify with Being Chronically Ill, Not Disabled

News Raxone Delays Assisted Ventilation in DMD Patients by 3 Years, Phase 3 Trial Results Show

News Axovant Enters Global Licensing Deal with Benitec for Investigational AXO-AAV-OPMD Program to Treat OPMD

News #PPMD2018 – Mexico Lags in Degree and Quality of Duchenne Care, Advocacy Groups Say

News Acceleron’s ACE-083 Earns Orphan Drug Status for Facioscapulohumeral MD

columns, You, Me and MD - a Column by Leah Leilani Treat Your Mental Health Before It Becomes Too Much to Bear

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