Families Of Sma

CURE SMA LEADS THE WAY TO A WORLD WITHOUT SPINAL MUSCULAR ATROPHY, THE NUMBER ONE GENETIC CAUSE OF DEATH FOR INFANTS. CURE SMA FUNDS AND DIRECTS COMPREHENSIVE RESEARCH THAT DRIVES BREAKTHROUGHS IN TREATMENT AND CARE AND PROVIDES FAMILIES THE SUPPORT THEY NEED FOR TODAY.

INDIVIDUALS WITH SMA NEED SPECIALIZED CARE AND EQUIPMENT, WHICH CAN PUT ENORMOUS LOGISTICAL AND FINANCIAL PRESSURE ON FAMILIES. CURE SMA IS COMMITTED TO MAKING SURE THAT FAMILIES HAVE THE BEST, MOST ACCURATE INFORMATION ABOUT SMA AND WHAT IT MEANS FOR THEM, AND TO MAKING SURE THAT THEY UNDERSTAND ALL THE OPTIONS AND RESOURCES AVAILABLE TO THEM FROM DAY-TO-DAY CARE TO THE CHANGING LANDSCAPE OF RESEARCH BREAKTHROUGHS AND TREATMENTS. THIS INCLUDES OUR CARE SERIES BOOKLETS, WHICH ARE SENT OUT AS PART OF INFORMATIONAL PACKETS AVAILABLE AT NO COST TO ANYONE WHO REQUESTS ONE. DEVELOPED BY MEDICAL EXPERTS AND REVIEWED BY FAMILIES, THESE BOOKLETS COVER TOPICS SUCH AS BREATHING, NUTRITION, PALLIATIVE CARE, GENETICS, MUSCULOSKELETAL ISSUES, AND MORE. IN ADDITION, OUR EQUIPMENT POOL GIVES FAMILIES ACCESS TO SPECIALIZED ITEMS LIKE CAR BEDS, STROLLERS AND BATHING SYSTEMS AT NO COST. EVERY NEWLY-DIAGNOSED FAMILY RECEIVES A CARE PACKAGE FROM CURE SMA, FULL OF TOYS APPROPRIATE TO THE CHILD'S TYPE OF THE DISEASE, IMPARTIAL INFORMATION FOR PARENTS, AND USEFUL ITEMS SUGGESTED BY OTHER SMA PARENTS BASED ON THEIR OWN EXPERIENCES. EVERY NEW ADULT TO THE COMMUNITY RECEIVES A PACKAGES OF SPECIAL INDEPENDENCE RELATED ITEMS.CURE SMA ALSO EDUCATES THE PUBLIC ABOUT SMA, TO STRENGTHEN THE SUPPORT AVAILABLE TO FAMILIES. THIS INCLUDES DIRECTIONS, A BIANNUAL FAMILY SUPPORT NEWSLETTER, AND COMPASS, A QUARTERLY UPDATE ON RESEARCH DEVELOPMENTS.CURE SMA WORKS DIRECTLY WITH CLINICIANS, HEALTH CARE PROVIDERS, AND CAREGIVERS TO ENSURE THAT PATIENTS HAVE ACCESS TO THE BEST POSSIBLE CARE.

CURE SMA FOCUSES ON THREE DIFFERENT YET EQUALLY CRITICAL AND INTERDEPENDENT RESEARCH AREAS: BASIC RESEARCH, TO UNDERSTAND THE CAUSE AND BIOLOGY OF SMA; DRUG DISCOVERY, TO CONVERT BASIC RESEARCH IDEAS INTO PRACTICAL NEW DRUG CANDIDATES; AND CLINICAL & REGULATORY RESEARCH, TO PROVIDE THE INFRASTRUCTURE TO TEST THE DRUG CANDIDATES. DURING THIS PERIOD CURE SMA FUNDED TENONGOING BASIC RESEARCH GRANTS, AS WELL AS TWO ONGOING DRUG DISCOVERY PROJECTS. WE ALSO FUNDED AND EXECUTED MULTIPLE CLINICAL AND REGULATORY FOCUSED PROJECTS, INCLUDING PROJECTS TO IMPLEMENT AND SUPPORT NEWBORN SCREENING FOR SMA AT THE STATE LEVEL, TO INCREASE THE NUMBER OF TRAINED SMA CLINICAL TRIALS SITES IN THE US, TO DEVELOP NEW OUTCOME MEASURES FOR SMA CLINICAL TRIALS, AND TO COLLECT DATA TO UNDERSTAND THE PATIENT EXPERIENCE WITH SMA. CURRENTLY, THERE ARE THREE APPROVED DRUGS FOR SMA, WITH ANOTHER SEVERAL DOZEN DRUG PROGRAMS IN DEVELOPMENT, INCLUDING SEVEN CLINICAL TRIALS. CURE SMA HAS FUNDED HALF OF ALL THE ONGOING DRUG PROGRAMS IN SMA.CURE SMA HAS EXPANDED FOCUS TO SUPPORT CLINICAL CARE. THE CURE SMA CARE CENTER NETWORK KICKED OFF IN 2018. IN 2022, THE SMA CARE CENTER NETWORK EXPANDED BY 5 ADDITIONAL CENTERS FOR A TOTAL OF 24 CENTERS WHO RECEIVED GRANT FUNDING. THESE CENTERS PROVIDE PATIENT CONSENTED ELECTRONIC MEDICAL RECORD DATA TO THE CURE SMA CLINICAL DATA REGISTRY. THE GOAL OF THE SMA CARE CENTER NETWORK AND CLINICAL DATA REGISTRY IS TO IMPROVE CLINICAL CARE FOR INDIVIDUALS WITH SMA BY ESTABLISHING AN EVIDENCE-BASED STANDARD OF CARE THAT WILL BE AVAILABLE AND DISTRIBUTED NATIONALLY. THE CLINICAL DATA REGISTRY HAS PROGRESSIVELY GROWN, AND MORE THAN 750 PATIENTS ARE ENROLLED. ADDITIONAL DATA ELEMENTS HAVE BEEN ADDED TO INCLUDE PROVIDER NAME AND SPECIALTY FOR EACH CLINICAL ENCOUNTER. CURE SMA COLLABORATES WITH STATE PUBLIC HEALTH LABS AND LEGISLATURES TO ADVOCATE FOR SMA NEWBORN SCREENING IMPLEMENTATION THROUGHOUT THE US. CURRENTLY 47 STATES SCREEN NEWBORNS FOR SMA FACILITATING EARLY PRESYMPTOMATIC TREATMENT. THIS REPRESENTS 97% OF ALL INFANTS BORN IN THE US. CURE SMA CO-CREATED FOUR NEW HEALTHCARE PROVIDER ENDURING CONTINUING MEDICAL EDUCATION PROGRAMS IN ADDITION TO THE IN-PERSON CURE SMA CLINICAL CARE SESSION OFFERED IN JUNE 2022. RESOURCES WERE ALSO DEVOTED TO MAINTAINING WEBSITE LISTINGS OF CARE CENTERS, SMA NEWBORN SCREENING REFERRAL CENTERS AND TREATMENT CENTERS THAT PROVIDE FDA-APPROVED SMA TREATMENTS.

WE HOSTED THE 2022 ANNUAL SMA CONFERENCE IN ANAHEIM, CA, WITH OVER 2,057 REGISTERED ATTENDEES. OUR ANNUAL CONFERENCE IS AN INVALUABLE RESOURCE FOR THE ENTIRE SMA COMMUNITY. THE CONFERENCE IS THE LARGEST IN THE WORLD FOCUSED SPECIFICALLY ON SMA. THE RESEARCHER AND CLINICAL CARE CONFERENCE ATTRACTED THE TOP SCIENTISTS AND COMPANIES IN THE FIELD. THE COMMUNITY CONFERENCE INCLUDES A VARIETY OF WORKSHOPS, KEYNOTE SESSIONS WITH LEADING EXPERTS, AND MORE-PLUS GREAT EVENTS SUCH AS OUR TEEN AND ADULTS WITH SMA SOCIALS, THE FAMILY FRIENDLY RESEARCHER POSTER SESSION AND OUR MEET & GREET WITH A FAMILY FUN FEST. THE RESEARCHER CONFERENCE CREATES OPEN COMMUNICATION OF EARLY, UNPUBLISHED SCIENTIFIC DATA, ACCELERATING THE PACE OF RESEARCH. THE MEETING ALSO FURTHERS RESEARCH BY BUILDING PRODUCTIVE COLLABORATIONS-INCLUDING CROSS-DISCIPLINARY DIALOGUE, PARTNERSHIPS, INTEGRATION OF NEW RESEARCHERS AND DRUG COMPANIES, AND EDUCATIONAL OPPORTUNITIES FOR JUNIOR RESEARCHERS. COMMUNITY ATTENDEES HAVE DIRECT ACCESS TO THE LATEST INFORMATION ABOUT RESEARCH, AND RESEARCHERS HAVE AN OPPORTUNITY TO BUILD PERSONAL CONNECTIONS WITH THE PATIENTS WHO WILL BENEFIT FROM THEIR WORK.