The Parent Project For Muscular Dystrophy Research Inc

END DUCHENNE MUSCULAR DYSTROPHY THROUGH RESEARCH, ADVOCACY, EDUCATION, & OPTIMAL MEDICAL CARE.

RESEARCHPPMD'S RESEARCH PROGRAM IDENTIFIES AND AGGRESSIVELY FUNDS THE MOST PROMISING NEAR AND LONG-TERM DUCHENNE RESEARCH ACROSS NUMEROUS THERAPEUTIC STRATEGIES; STIMULATES NEW RESEARCH TO ENSURE THAT THE THERAPEUTIC PIPELINE IS RICH WITH OPPORTUNITY; FACILITATES PRE-COMPETITIVE INDUSTRY INTERACTIONS THROUGH THE DUCHENNE DRUG DEVELOPMENT ROUNDTABLE TO ENHANCE AND HARMONIZE THE CLINICAL TRIAL LANDSCAPE; AND SUPPORTS MULTI-STAKEHOLDER DATA MODELING CONSORTIA TO ACCELERATE DRUG DEVELOPMENT TIMELINES. IN ADDITION TO SIGNIFICANT PRE-CLINICAL AND ACADEMIC RESEARCH FUNDING, THROUGH THE VENTURE PATHWAYS PROGRAM, PPMD MAKES INVESTMENTS IN EARLY-STAGE BIOPHARMACEUTICAL COMPANIES TO CATALYZE DEVELOPMENT OF NOVEL THERAPIES TO TREAT DUCHENNE AND BECKER. THE DUCHENNE REGISTRYTHE DUCHENNE REGISTRY IS A PATIENT-REPORTED REGISTRY FOR INDIVIDUALS WITH DUCHENNE AND BECKER MUSCULAR DYSTROPHY AND CARRIER FEMALES, ESTABLISHED BY PPMD IN 2007, THE REGISTRY WAS CREATED TO BRIDGE THE INFORMATION GAP BETWEEN CLINICIANS, RESEARCHERS, AND THE PATIENT COMMUNITY, THEREBY ADDRESSING MEDICAL CARE NEEDS AND ACCELERATING THE PACE OF THERAPEUTIC ADVANCEMENTS. OVER 5,000 FAMILIES ACROSS THE GLOBE HAVE EMBRACED THEIR ROLE AS CITIZEN SCIENTISTS AND HAVE CONTRIBUTED TO THE LARGEST PATIENT-REPORTED DATA SET FOR DUCHENNE.SINCE ITS LAUNCH, DATA HAS BEEN REFERENCED IN THIRTEEN PUBLICATIONS AND EXPORTED BY RESEARCHERS 65 TIMES. THE REGISTRY HAS ALSO BEEN USED TO IDENTIFY AND CONNECT PEOPLE WITH DUCHENNE TO OVER 80 ACTIVELY RECRUITING TRIALS. THE DUCHENNE OUTCOMES RESEARCH INTERCHANGE (THE INTERCHANGE): THE INTERCHANGE COLLECTS PATIENT DATA AND CLINICIAN ENTERED DATA SO BOTH SETS ARE SO BOTH AGGREGATED AND ANALYZED IN ONE PLACE AND KEY INDUSTRY STAKEHOLDERS CAN GET A FULL PICTURE OF HOW DIFFERENT PEOPLE LIVE WITH DUCHENNE. THE ELECTRONIC HEALTH RECORD (EHR) STUDY: THE EHR STUDY EXTRACTS DATA FROM SEVERAL OF PPMD'S CERTIFIED DUCHENNE CARE CENTERS ACROSS THE COUNTRY TO IMPROVE CARE AND ANSWER RESEARCH QUESTIONS IN A WAY THAT DOES NOT REQUIRE EXTRA EFFORT FROM PATIENTS OR THEIR DOCTOR.THE REGISTRY IS ALSO HOME TO PPMD'S ELECTRONIC HEALTH RECORD (EHR) EXTRACTION STUDY WHICH RECEIVES CORE CLINICAL DATA ELEMENTS FROM SOME CARE CENTERS. CAREPPMD'S CARE PROGRAM STRIVES TO ENSURE THAT PEOPLE LIVING WITH DUCHENNE ARE LIVING LONGER, STRONGER LIVES, BY HELPING PROVIDE ACCESS TO EXPERT HEALTHCARE PROVIDERS, A COMPREHENSIVE TEAM OF SUB-SPECIALISTS, AND APPROVED TREATMENTS. PPMD IDENTIFIES GAPS IN CARE FOR PEOPLE WITH DUCHENNE THROUGHOUT THE LIFESPAN AND COLLABORATES WITH HEALTH CARE PROFESSIONALS ACROSS THE GLOBE TO IMPROVE HEALTH OUTCOMES FOR ALL DUCHENNE PATIENTS. PPMD'S CERTIFIED DUCHENNE CARE CENTER PROGRAM (CDCC) HELPS TO ENSURE THAT CENTERS COMPLY WITH THE STANDARDS OF CARE AND SERVICES THAT HAVE BEEN ESTABLISHED IN THE DUCHENNE CARE GUIDELINES. ALL CERTIFIED DUCHENNE CARE CENTERS HAVE MET THE REQUIREMENTS FOR, AND AGREE TO PROVIDE, STANDARDIZED CARE AND SERVICES.

EDUCATIONPPMD'S EDUCATION INITIATIVES INCREASE AWARENESS AND UNDERSTANDING OF THE DISEASE PROGRESSION AND THE COMPONENTS OF MULTIDISCIPLINARY COMPREHENSIVE CARE NECESSARY TO OPTIMIZE QUALITY AND QUANTITY OF LIFE THROUGHOUT THE LIFESPAN. PPMD PARTNERS WITH EXPERTS IN DUCHENNE TO DEVELOP UP-TO-DATE INFORMATIONAL RESOURCES REGARDING TREATMENT AND CARE THAT WE THEN SHARE BROADLY. PPMD COLLABORATES WITH NATIONAL AND INTERNATIONAL ORGANIZATIONS TO DEVELOP AND DISSEMINATE EDUCATIONAL RESOURCES APPROPRIATE FOR THE GLOBAL DUCHENNE COMMUNITY.ENGAGEMENT PPMD BRINGS PEOPLE TOGETHER FOR SUPPORT AND LEARNING OPPORTUNITIES AT ALL TIMES. PPMD HOSTS THE LARGEST GLOBAL DUCHENNE CONFERENCE EVERY SUMMER, BRINGING TOGETHER PATIENTS, FAMILIES, CLINICIANS, RESEARCHERS, AND INDUSTRY PARTNERS FROM ACROSS THE GLOBE TO SHARE THE LATEST NEWS AND UPDATES. PPMD ALSO CONVENES MEMBERS OF THE DUCHENNE COMMUNITY THROUGH DIFFERENT GROUP GATHERINGS - VIRTUALLY AND IN PERSON AT VARIOUS POINTS THROUGHOUT THE YEAR. CURRENT GROUPS INCLUDE DUCHENNE CARRIERS, DUCHENNE SIBLINGS, DUCHENNE DADS, DUCHENNE GRANDPARENTS AND NEWLY DIAGNOSED GROUPS. PPMD ALSO OFFERS 1:1 CONNECTION BASED ON SPECIFIC NEEDS THROUGH PPMD TOGETHER - VIRTUAL RESOURCE MEETINGS.

ADVOCACYPPMD'S ADVOCACY EFFORTS ARE FOCUSED ON ADVANCING CARE AND TREATMENTS FOR DUCHENNE BY LEVERAGING FEDERAL RESOURCES, BUILDING PARTNERSHIPS, AND ADVANCING REGULATORY PROCEDURES AND INFRASTRUCTURE. WE EMPOWER ADVOCATES WITH THE TOOLS AND INFORMATION TO ENGAGE WITH CONGRESSIONAL REPRESENTATIVES TO FOSTER DUCHENNE CHAMPIONS WITHIN CONGRESS AND TO SUPPORT LEGISLATION AND REGULATORY POLICIES, ENSURING THAT DUCHENNE PRIORITIES ARE REFLECTED ACROSS ALL GOVERNMENT AGENCIES.PPMD ALSO LEADS EFFORTS FOR FEDERAL AND LOCAL LEGISLATION TO SUPPORT NEWBORN SCREENING FOR DUCHENNE.