THE PARENT PROJECT FOR MUSCULAR DYSTROPHY RESEARCH INC

RESEARCHPPMD'S RESEARCH PROGRAM IDENTIFIES AND FUNDS THE MOST PROMISING NEAR- AND LONG-TERM THERAPEUTIC STRATEGIES FOR DUCHENNE AND BECKER MUSCULAR DYSTROPHY. WE INVEST ACROSS THE DEVELOPMENT PIPELINE-FROM BASIC SCIENCE TO CLINICAL APPLICATION-WHILE ACTIVELY STIMULATING NEW RESEARCH TO KEEP THE THERAPEUTIC PIPELINE ROBUST. THROUGH INITIATIVES LIKE THE DUCHENNE DRUG DEVELOPMENT ROUNDTABLE, WE FOSTER PRE-COMPETITIVE INDUSTRY COLLABORATION TO HARMONIZE THE CLINICAL TRIAL LANDSCAPE AND ACCELERATE PROGRESS.IN ADDITION TO FUNDING ACADEMIC AND PRE-CLINICAL RESEARCH, OUR VENTURE PATHWAYS PROGRAM INVESTS IN EARLY-STAGE BIOPHARMACEUTICAL COMPANIES TO DRIVE THE DEVELOPMENT OF NOVEL TREATMENTS FOR DUCHENNE AND BECKER MUSCULAR DYSTROPHY.THE DUCHENNE REGISTRYESTABLISHED IN 2007, THE DUCHENNE REGISTRY IS THE LARGEST PATIENT-REPORTED DATA REPOSITORY FOR INDIVIDUALS LIVING WITH DUCHENNE AND BECKER MUSCULAR DYSTROPHY AND CARRIER FEMALES. WITH PARTICIPATION FROM OVER 6,000 FAMILIES WORLDWIDE, THE REGISTRY HELPS BRIDGE INFORMATION GAPS BETWEEN FAMILIES, CLINICIANS, AND RESEARCHERS. IT HAS CONTRIBUTED TO 14 PEER-REVIEWED PUBLICATIONS AND SUPPORTED OVER 85 CLINICAL TRIALS THROUGH TARGETED OUTREACH.PPMD ALSO MANAGES COMPLEMENTARY INITIATIVES:-THE DUCHENNE OUTCOMES RESEARCH INTERCHANGE (THE INTERCHANGE) AGGREGATES PATIENT- AND CLINICIAN-REPORTED DATA TO PROVIDE A COMPREHENSIVE VIEW OF THE LIVED EXPERIENCE OF DUCHENNE, SUPPORTING REAL-WORLD EVIDENCE GENERATION.- THE ELECTRONIC HEALTH RECORD (EHR) STUDY EXTRACTS DE-IDENTIFIED CLINICAL DATA FROM CERTIFIED DUCHENNE CARE CENTERS TO IMPROVE CARE AND INFORM RESEARCH WITHOUT ADDING BURDEN TO PATIENTS OR PROVIDERS.CAREPPMD'S CARE PROGRAM ENSURES INDIVIDUALS WITH DUCHENNE HAVE ACCESS TO EXPERT CLINICAL TEAMS, APPROVED THERAPIES, AND COMPREHENSIVE MULTIDISCIPLINARY CARE ACROSS THEIR LIFESPAN. WE IDENTIFY GAPS IN CARE AND WORK COLLABORATIVELY WITH GLOBAL HEALTHCARE PROVIDERS TO CLOSE THOSE GAPS AND IMPROVE OUTCOMES.OUR CERTIFIED DUCHENNE CARE CENTER (CDCC) PROGRAM CERTIFIES CARE CENTERS THAT MEET RIGOROUS STANDARDS ALIGNED WITH THE DUCHENNE CARE GUIDELINES. THESE CENTERS COMMIT TO DELIVERING CONSISTENT, HIGH-QUALITY CARE BASED ON BEST PRACTICES.

EDUCATIONPPMD'S EDUCATION INITIATIVES EQUIP FAMILIES, HEALTHCARE PROVIDERS, AND THE BROADER COMMUNITY WITH THE INFORMATION NEEDED TO NAVIGATE DUCHENNE CARE ACROSS ALL STAGES OF THE DISORDER. WE PARTNER WITH CLINICAL AND SCIENTIFIC EXPERTS TO DEVELOP ACCURATE, ACCESSIBLE RESOURCES AND SHARE THEM BROADLY. WE ALSO COLLABORATE WITH DOMESTIC AND INTERNATIONAL ORGANIZATIONS TO ENSURE EDUCATIONAL MATERIALS REACH GLOBAL AUDIENCES.ENGAGEMENTPPMD FOSTERS A STRONG, INFORMED, AND CONNECTED COMMUNITY. EACH SUMMER, WE HOST THE WORLD'S LARGEST DUCHENNE CONFERENCE, BRINGING TOGETHER FAMILIES, CLINICIANS, RESEARCHERS, AND INDUSTRY PARTNERS TO SHARE UPDATES AND BUILD CONNECTIONS.THROUGHOUT THE YEAR, WE OFFER VIRTUAL AND IN-PERSON GATHERINGS FOR SUBGROUPS INCLUDING DUCHENNE CARRIERS, SIBLINGS, DADS, GRANDPARENTS, AND NEWLY DIAGNOSED FAMILIES.THROUGH PPMD TOGETHER, WE ALSO PROVIDE PERSONALIZED ONE-ON-ONE VIRTUAL SUPPORT TO HELP FAMILIES NAVIGATE THEIR INDIVIDUAL CHALLENGES.