Cure Cmd Inc

2022 CONGENITAL MUSCLE DISEASE SCIENTIFIC SYMPOSIUM - CURE CMD (CURE CONGENITAL MUSCULAR DYSTROPHY) SEEKS SUPPORT FROM THE NATIONAL INSTITUTES OF HEALTH TO COLLABORATE WITH ALIGNED PATIENT ADVOCACY ORGANIZATIONS A FOUNDATION BUILDING STRENGTH (AFBS, FOCUSED ON NEMALINE MYOPATHY OR NM) AND TEAM TITIN (FOCUSED ON TITINOPATHY OR TTN) FOR THE 2022 CONGENITAL MUSCLE DISEASE SCIENTIFIC SYMPOSIUM, TO BE HELD JUNE 30 - JULY 3, 2022. WITH SIGNIFICANT OVERLAP IN SYMPTOMATOLOGY, CARE MANAGEMENT, AND RESEARCH COMMUNITIES, REPRESENTATIVES FROM EACH ORGANIZATION BELIEVE ATTENDEES WILL BENEFIT SIGNIFICANTLY FROM INFORMATION EXCHANGE AND COLLABORATION. THE ULTIMATE AIM OF THE MEETING IS TO ACCELERATE PROGRESS TOWARD CLINICAL TRIALS, TREATMENTS, AND EVENTUALLY, A CURE FOR CONGENITAL MUSCLE DISORDERS. THE 2022 EVENT WOULD BE THE SECOND MEETING OF THIS TYPE, AND IN THE TWO YEARS SINCE THE MEETING, MULTIPLE ADVANCEMENTS HAVE BEEN MADE FOR EACH OF THE THREE CONDITIONS REPRESENTED AT THE CONFERENCE. KEY OBJECTIVES ARE TO: - INTRODUCE NEW CONGENITAL MUSCLE DISEASE-FOCUSED RESEARCH PROJECTS. THE HOST PATIENT ADVOCACY GROUPS ARE FUNDING 13 NEW RESEARCH PROJECTS SINCE 2019’S IN-PERSON CONVENING; - DISCUSS NEXT STEPS FOR CREATING INNOVATIVE PLATFORMS LIKE THE NCATS-LED PLATFORM VECTOR GENE THERAPY (PAVE-GT), TAKING ADVANTAGE OF COMMON MUTATION CLASSES AND PATHOPHYSIOLOGICAL PATHWAYS ACROSS CMD SUBTYPES; - ASSESS AND UPDATE CURRENT SUBTYPE-SPECIFIC RESEARCH PLANS AND PRIORITIES FOR EACH DISEASE, CURRENT SINCE 2019, AND HIGHLIGHT DEFICIENCIES IN EACH ON THE PATH TO CLINICAL TRIALS; - INCREASE COMMUNICATION AMONG RESEARCHERS OF RARE NEUROMUSCULAR DISEASE. THOUGH THESE DISEASES ARE DISTINCT FROM A HISTOLOGICAL AND GENETIC STANDPOINT, MANY TREATMENT MODALITIES AND INTERVENTIONS WILL BE SIMILAR IN NATURE. INFORMATION-SHARING IS KEY; - DISCUSS RESEARCH AND OPPORTUNITIES FOR SCIENTIFIC ADVANCEMENT AROUND CARDIAC AND PULMONARY FUNCTION, TWO SYSTEMS THAT HAVE THE GREATEST IMPACT ON HEALTH AND QUALITY OF LIFE FOR THE CONGENITAL MUSCLE DISEASE COMMUNITY; - CREATE, SHAPE, AND REFINE RESEARCH PROJECTS AND PLANS WITH CLEAR PATIENT INPUT ON NEED AND DESIGN. DISCUSSIONS WITH AFFECTED INDIVIDUALS AND CLINICIANS WILL HELP ENSURE THAT RESEARCH IS RESPONSIVE TO THE COMMUNITY'S NEEDS. WITH CURE CMD, AFBS, AND TEAM TITIN ALL WORKING WITH RESEARCHERS AND INDUSTRY TO SPEED TOWARD CLINICAL TRIALS, THESE COMMUNITIES ARE PRIMED FOR THE NEXT COLLABORATIVE MEETING.

2019 CONGENITAL MUSCLE DISEASE SCIENTIFIC SYMPOSIUM