FAMILIES OF SPINAL MUSCULAR ATROPHY
925 BUSSE RD, ELK GROVE VILLAGE, IL 60007 www.curesma.org

---

Organizations Filed Purposes: CURE SMA FUNDS AND DIRECTS THE LEADING SMA RESEARCH PROGRAMS TO DEVELOP A TREATMENT AND CURE FOR THE DISEASE. THE SUCCESSFUL RESULTS AND PROGRESS THAT THE ORGANIZATION HAS DELIVERED, FROM BASIC RESEARCH TO DRUG DISCOVERY TO CLINICAL TRIALS, PROVIDE REAL HOPE FOR FAMILIES AND PATIENTS IMPACTED BY THE DISEASE. THE CHARITY HAS INVESTED OVER $60 MILLION IN RESEARCH AND HAS BEEN INVOLVED IN FUNDING HALF OF ALL THE ONGOING NOVEL DRUG PROGRAMS FOR SMA. CURE SMA IS A NONPROFIT 501(C)(3) ORGANIZATION, WITH 34 CHAPTERS AND 115,000 MEMBERS AND SUPPORTERS THROUGHOUT THE UNITED STATES, AND IS DEDICATED TO CREATING A TREATMENT AND CURE BY FUNDING AND ADVANCING A COMPREHENSIVE RESEARCH PROGRAM, SUPPORTING SMA FAMILIES THROUGH NETWORKING, INFORMATION AND SERVICES, IMPROVING CARE FOR ALL SMA PATIENTS, EDUCATING HEALTHCARE PROFESSIONALS AND THE PUBLIC ABOUT SMA, ENLISTING GOVERNMENT SUPPORT FOR SMA, EMBRACING ALL TOUCHED BY SMA IN A CARING COMMUNITY.

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 EMOTIONAL, 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. 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.CURE SMA ALSO EDUCATE 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, MEDICAL DOCTORS, SPECIALISTS, AND SKILLED 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 AND REGULATORY RESEARCH, TO PROVIDE THE INFRASTRUCTURE TO TEST THE DRUG CANDIDATES. DURING THIS PERIOD CURE SMA FUNDED 16 NEW AND ONGOING BASIC RESEARCH GRANTS, AS WELL AS TWO ONGOING AND TWO NEW DRUG DISCOVERY PROJECTS. WE ALSO FUNDED AND EXECUTED MULTIPLE CLINICAL AND REGULATORY FOCUSED PROJECTS, INCLUDING PROJECTS TO IMPLEMENT NEWBORN SCREENING FOR SMA AT THE STATE LEVEL, TO INCREASE THE NUMBER OF TRAINED SMA CLINICAL TRIALS SITES IN THE US, AND TO UNDERSTAND THE PATIENT EXPERIENCE WITH SMA. CURRENTLY, THERE ARE TWO APPROVED DRUGS FOR SMA, WITH ANOTHER 20 DRUG PROGRAMS IN DEVELOPMENT, INCLUDING SIX IN 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 SMA CARE CENTER NETWORK KICKED OFF IN 2018 AND 14 CENTERS 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. CURE SMA ALSO PROVIDES PATIENT AND HEALTHCARE PROVIDER EDUCATION, WITH EXPERTISE FROM ITS MEDICAL ADVISORY COUNCIL. RESOURCES WERE ALSO DEVOTED TO MAINTAIN WEBSITE LISTINGS OF CARE CENTERS THAT PROVIDE FDA-APPROVED THERAPIES FOR PATIENTS.

WE HOSTED OUR 32ND ANNUAL SMA CONFERENCE VIRTUALLY, WITH OVER 6,600 REGISTERED TO ATTEND. 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 FAMILY CONFERENCE INCLUDES A VARIETY OF WORKSHOPS, KEYNOTE SESSIONS WITH LEADING EXPERTS, AND MORE-PLUS FUN EVENTS SUCH AS OUR ADULTS WITH SMA SOCIALS AND VIRTUAL LOUNGES, GRANDPARENTS VIRTUAL SOCIAL AND A CELEBRATION CEREMONY TO END THE CONFERENCE. 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. FAMILIES 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.

---

---

Data for this page was sourced from XML published by IRS (public 990 form dataset) from: https://s3.amazonaws.com/irs-form-990/202043159349303769_public.xml