Specifically Named Diseases

"Stephen Heywood was diagnosed with ALS at age 29. After finding no treatment options, his brother, Jamie Heywood, realized that a gap existed in the preclinical development of therapies for ALS. In 1999, Jamie founded the ALS Therapy Development Foundation, now the ALS Therapy Development Institute (ALS TDI), in the basement of his family's Newton, MA home in an effort to bridge this gap and bring effective treatments to people with ALS as quickly as possible." - https://www.als.net/about-als-tdi/

The Parkinson’s Foundation makes life better for people with Parkinson’s disease by improving care and advancing research toward a cure. In everything we do, we build on the energy, experience and passion of our global Parkinson’s community.

TO PROVIDE LONG TERM DIABETES MELLITUS RESEARCH IN CONJUNCTION WITH A HOSPITAL.

TB ALLIANCE IS A NOT-FOR-PROFIT ORGANIZATION DEDICATED TO THE DISCOVERY, DEVELOPMENT AND DELIVERY OF BETTER, FASTER-ACTING AND AFFORDABLE TUBERCULOSIS DRUGS THAT ARE AVAILABLE TO THOSE WHO NEED THEM.

TO ADVANCE KNOWLEDGE OF, AND CARE FOR PEOPLE WITH BLADDER CANCER.

THE ORGANIZATION SUPPORTS AND ENCOURAGES CLINICAL INVESTIGATIONS AND MEDICAL RESEARCH IN COLLABORATION WITH THE UNIV. OF FL COLLEGE OF MEDICINE CONDUCTED THROUGHOUT THE WORLD FOR TREATMENT & CURE OF REFLEX SYMPATHETIC DYSTROPHY (RSD) & COMPLEX REGIONAL PAID SYNDROME (CRPS).

AAHI IS A NONPROFIT BIOTECH RESEARCH INSTITUTE, BRINGING TOGETHER THE BEST EXPERTS, TECHNOLOGIES, AND PLATFORMS TO CREATE ACCESSIBLE, HIGH-QUALITY PRODUCTS AND SOLUTIONS FOR HARNESSING THE IMMUNE SYSTEM TO ALLEVIATE DISEASE.

ADVANCING HEALTH AND WELLBEING WORLDWIDE

TO HELP FIND THE CURE FOR DIABETES, AND UNTIL THAT GOAL IS ACHIEVED, TO PROVIDE THE CARE AND SELF-MANAGEMENT SKILLS NEEDED TO COMBAT THE LIFE-THREATENING COMPLICATIONS OF DIABETES, AND TO PROMOTE PUBLIC EDUCATION ABOUT THE CAUSES, PREVENTION AND TREATMENT OF THE DISEASE.

TO BE THE PREEMINENT SOURCE OF MEDICAL-GRADE PORCINE TISSUE UTILIZED TO CURE DISEASES, WITH THE PRIMARY COMMITMENT TO DIABETES.

PROTECT AND ENHANCE THE HEALTH OF THE U.S. SWINE HERD THROUGH COORDINATED GLOBAL RESEARCH MONITORING, TARGETED RESEARCH INVESTMENTS THAT MINIMIZE THE IMPACT OF FUTURE DISEASE THREATS, AND ANALYSIS OF SWINE HEALTH DATA.

TO SUPPORT ALS RESEARCH AND SCIENTIFIC INVESTIGATIONS AT THE UNIVERSITY OF MASSACHUSETTS CHAN MEDICAL SCHOOL BY RAISING FUNDS THROUGH EVENTS, CAMPAIGNS, FOUNDATION GRANTS AND NUMEROUS OTHER COMMUNITY OUTREACH ACTIVITIES, TO AID IN FINDING A CAUSE, TREATMENT AND CURE FOR ALS.

ACCELERATING THE DEVELOPMENT OF ADVANCED DIAGNOSTIC TOOLS AND EFFECTIVE TREATMENTS FOR SYSTEMIC AMYLOIDOSIS THROUGH COLLABORATION AND INNOVATION.

TO FUND RESEARCH AND PUBLIC AWARENESS FOCUSED ON DEVELOPING A CURE FOR EOSINOPHILIC DISORDER

TO PROVIDE NEW MEXICO CANCER PATIENTS AND THEIR FAMILIES ACCESS TO THE LATEST RESEARCH, TECHNOLOGIES, AND CLINICAL SERVICES IN PREVENTION, SCREENING, DETECTION, DIAGNOSIS/STAGING, TREATMENT, SUPPORTIVE CARE, AND CONTINUED SURVEILLANCE THROUGH COLLABORATION AMONG PUBLIC AND PRIVATE HEALTHCARE PROVIDERS.

To advocate for and fund research directed towards a cure or treatment options for patients with Sanfilippo Syndrome

TO PROVIDE GLOBAL EDUCATIONAL INFORMATION, SERVICES AND SUPPORT TO THOSE AFFECTED BY ESSENTIAL TREMOR, AND TO HEALTH CARE PROVIDERS, WHILE PROMOTING AND FUNDING ET RESEARCH.

TO PROVIDE ASSISTANCE WITH DIABETES AND OBESITY EDUCATION, PREVENTION, MANAGEMENT AND RESEARCH.

Let Every Woman Know - Alaska is dedicated to raising awareness, sharing information, and saving lives through local education of all Alaskans and support for those women.

THE ORGANIZATION'S MISSION IS TO SAVE THE LIVES OF CHILDREN AND YOUNG ADULTS WHO ARE GENETICALLY PREDISPOSED OR OTHERWISE SUSCEPTIBLE TO SUDDEN DEATH TO CARDIAC ARRHYTHMIAS AND TO PROVIDE EDUCATION AND SUPPORT TO FAMILIES AND THE MEDICAL COMMUNITY WHO ARE DEALING WITH THESE DISORDERS. THE SADS FOUNDATION IS DEDICATED TO PROVIDING INFORMATION, ASSISTANCE, AND HOPE.

TO INCREASE AWARENESS, IDENTIFICATION, UNDERSTANDING, AND TREATMENT OF CHILDREN WITH APRAXIA, DYSLEXIA, AND SEX CHROMOSOME DISORDERS AND TO FOSTER AND PROMOTE SCIENTIFIC RESEARCH RELATING TO THESE DISORDERS.

APFED IS DEDICATED TO PREVENTING AND CURING EOSINOPHIL-ASSOCIATED DISEASES WHILE ENHANCING THE QUALITY OF LIFE FOR PATIENTS AND FAMILIES. WE ACHIEVE THIS BY PASSIONATELY EMBRACING, SUPPORTING, AND ADVANCING EDUCATION, AWARENESS, RESEARCH, ADVOCACY, AND SUPPORT INITIATIVES FOR T

TO CARRY ON AND SUPPORT SCIENTIFIC RESEARCH, HEALTH SYSTEMS DEVELOPMENT, STRENGTHENING AND IMPLEMENTATION, HEALTH SCIENCE EDUCATION AND DEVELOPMENT AND HEALTH COUSULTING, IN THE FIELD OF POVERTY-RELATED COMMUNICABLE AND NON-COMMUNICABLE DISEASES (WITH A PARTICULAR EMPHASIS ON HIV/AIDS AND TUBERCULOSIS), FOR THE BENEFIT OF POOR, DISTRESSED, UNDER-PRIVILEGED AND VULNERABLE COMMUNITIES AND SOCIETIES AROUND THE WORLD, AND ANY OTHER ACTIVITIES RELATED THERETO.

TO FUND RESEARCH TO HELP THE MEDICAL COMMUNITY BETTER UNDERSTAND ADDICTION DISORDERS BOTH ON THE PSYCHOLOGICAL AND PHYSIOLOGICAL LEVELS.

THE INTERNATIONAL SOCIETY OF MANNOSIDOSIS AND RELATED DISEASES IS AN INTERNATIONALLY FOCUSED ORGANIZATION WHOSE MISSION IS TO ADVOCATE FOR FAMILIES AFFECTED BY ONE OF THE GLYCOPROTEIN STORAGE DISEASES:ALPHA MANNOSIDOSIS, ASPARTYLGLUCOSAMINURIA, BETA-MANNOSIDOSIS, FUCOSIDOSIS, GALACTOSIALIDOSIS,MUCOLIPIDOSIS II (I-CELL DISEASE), MUCOLIPIDOSIS III (PSEUDO-HURIER POLYDYSTROPHY), SCHINDLER DISEASE AND SIALIDOSIS. THESE DISEASES ARE THE ULTRA-ORPHANS OF THE LYSOSOMAL STORAGE DISEASE FAMILY, WHICH ENCOMPASS OVER 40 SIMILAR DISORDERS, AND CURRENTLY HAVE FEW TREATMENTS BEYOND SYMPTOMATIC CARE.ISMRD'S ADVOCACY EFFORTS ARE NOT LIMITED BY BORDERS, LANGUAGE, RACE OR RELIGION.

THE MISSION OF THE MATTHEW AND ANDREW AKIN FOUNDATION IS TO SUPPORT THE WORLD-RENOWNED EXPERTS AT THE HLH CENTER OF EXCELLENCE AT CINCINNATI CHILDREN'S HOSPITAL AND THEIR CORE PRIORITIES OF RESEARCH, EDUCATION AND FAMILY SUPPORT. WE ALSO GIVE BACK TO THE ORGANIZATIONS THAT SUPPORTED OUR FAMILY DURING OUR JOURNEY IN AN EFFORT TO MAKE A DIFFERENCE FOR THOSE THAT WILL BE FOLLOWING IN OUR FOOTSTEPS IN THE FUTURE.

THE ORGANIZATION IS WORKING TOWARDS HELPING FIND CURES AND/OR EFFECTIVE TREATMENTS FOR ORPHAN (RARE) DISEASES, AND TO FACILITATE RESEARCH OF SUCH DISEASES.

THE LYME DISEASE RESEARCH FOUNDATION, INC. IS A PUBLIC NON-PROFIT ORGANIZATION DEDICATED TO RESEARCH INTO THE UNDERSTANDING AND CARE OF PATIENTS WITH PERSISTENT SYMPTOMS OF LYME DISEASE.

RHIZOKIDS INTERNATIONAL WILL SECURE FUNDS TO SUPPORT RHIZOMELIC CHONDRODYSPLASIA PUNCTATA (RCDP) RESEARCH PROGRAMS, PROVIDE EDUCATION AND PROMOTE AWARENESS OF RCDP, AND SUPPORT RCDP FAMILIES.

SLC6A1 CONNECT IS A PATIENT ADVOCACY GROUP DEDICATED TO IMPROVING THE LIVES OF CHILDREN AND FAMILIES AFFECTED BY THE SLC6A1 MUTATION. OUR FOCUS IS TO RAISE AWARENESS AND FUNDRAISING TO ADVANCE SCIENTIFIC RESEARCH THAT WILL ULTIMATELY RESULT IN A CURE.

It is the mission of the MECP2 Duplication Foundation to provide families diagnosed with MECP2 duplication syndrome information and support, to increase awareness of the disorder, and to provide funding to cutting edge research that will one day treat those affected and enhance their quality of life.

SILENT SPRING INSTITUTE IS DEDICATED TO ADVANCING SCIENCE ON THE INFLUENCE OF ENVIRONMENTAL CHEMICALS ON WOMEN'S HEALTH, WITH A FOCUS ON BREAST CANCER PREVENTION.

The organization is committed to advancing scientific research and improving the lives of individuals affected by Neurofibromatosis NF1.

CRF is committed to finding a cure through an aggressively funded research agenda. CRF awards research grants bi-annually to accelerate the research process and to ensure that there is never a gap in funding new cutting-edge research projects. CRF is dedicated to improving the quality of life for cystinosis patients and to finding better treatments, including a cure, for our adults and children living with cystinosis.

TO PROVIDE PATIENT SUPPORT AND EDUCATION, RAISE PUBLIC AWARENESS, FUND RESEARCH, AND IMPROVE HEALTH CARE PRACTICES, REDUCING ILLNESS AND DEATH CAUSED BY PREECLAMPSIA AND HYPERTENSIVE DISORDERS OF PREGNANCY.

SPIROVATION PROVIDES MEDICAL AND CLINICAL RESEARCH SERVICES TO COMMERCIAL PHARMACEUTICAL AND BIOTECHNOLOGY COMPANIES.

The mission of the Phelan-McDermid Syndrome Foundation is to make today better and the future brighter for everyone living with Phelan-McDermid syndrome - from the moment of diagnosis to the delivery of treatments and cures.

THE MISSION OF NORTH EAST ALS CONSORTIUM (NEALS) IS TO TRANSLATE SCIENTIFIC ADVANCES INTO CLINCICAL RESEARCH AND NEW TREATMENTS FOR PEOPLE WITH AMYOTROPHIC LATERAL SCLEROSIS (ALS) AND MOTOR NEURON DISEASE (MND) AS RAPIDLY AS POSSIBLE.

THE MISSION OF MILA'S MIRACLE FOUNDATION IS TO FIND AND FUND PATHS TO A CURE FOR BATTEN AND OTHER LIFE-THREATENING NEUROLOGICAL DISORDERS. FAMILIES IN THIS COMMUNITY HAVE BEEN PIVOTAL IN RAISING THE NECESSARY FUNDS AND AWARENESS. TOGETHER, WITH OUR INCREDIBLE SCIENTISTS AND PARTNER ORGANIZATIONS, WE HOPE THAT OUR WORK WILL IMPROVE THE PROGNOSIS OF BATTEN DISEASE AND OTHER NEURODEGENERATIVE DISEASES. THROUGH OUR FUNDRAISING, WHICH INCLUDES GRASSROOTS EFFORTS, INDIVIDUALS AND FAMILY FOUNDATIONS, AND EVENTS, OUR FOUNDATION'S WORK HAS FOCUSED ON FIVE PRIMARY PROGRAM INITIATIVES TO DATE: AN ANTISENSE OLIGONUCLEOTIDE (ASO) CLINICAL TRIAL; A CLN7 GENE THERAPY TRIAL; BASIC SCIENCE RESEARCH FOR BATTEN DISEASE; A BATTEN & NEURODEGENERATIVE DISEASE PROGRAM CLINIC; AND A PEDIATRIC CELL ATLAS OF BATTEN PATHOBIOLOGY AND THERAPEUTIC RESPONSE. ADDITIONALLY, WE ARE FOCUSED ON HELPING TO GROW THE NEW FIELD OF TRULY PERSONAL MEDICINES, WHICH MILA'S STORY HAS OPENED UP. OUR HOPE IS TO GO FROM "MILA TO MIL

THE ORGANIZATION'S MISSION IS TO PROVIDE RESEARCH GRANTS TO FUND AGGRESSIVE RESEARCH INTO TREATMENTS FOR DUCHENNE MUSCULAR DYSTROPHY AND TO SUPPORT THE EFFORT TO TREAT, AND ULTIMATELY CURE, THIS DISEASE.

Behavioral Diabetes Institute is dedicated to helping people with diabetes live long, healthy, and happy lives. We provide clinical services, professional services for health care professionals and conduct research programs.

The primary purpose of the organization is the funding of research to find a cure for diabetes.

THE MISSION OF THE LYMAN MEDICAL RESEARCH FOUNDATION, INC. IS TO CONDUCT AND SUPPORT MEANINGFUL MEDICAL RESEARCH. INITIATED STUDIES STRIVE TOWARD IMPROVING PATIENT CARE AND HEALTHCARE PRACTICES. THE STUDIES ARE PUBLISHED AND SHARED WITH THE GREATER SCIENTIFIC AND MEDICAL COMMUNITY FOR THE GOOD OF THE PUBLIC. OUR MISSION IS TO KEEP LIFE ACTIVE THROUGH INNOVATIVE ORTHOPEDIC RESEARCH, EDUCATION AND PATIENT-CENTERED CARE.

OUR MISSION IS TO PERFORM INTEGRATIVE SPORTS RESEARCH WITH YOUNG ATHLETES TO MAINTAIN AND PROMOTE THEIR ATHLETIC PERFORMANCE, HEALTH, SAFETY AND QUALITY OF LIFE.

TO BE THE PHILANTHROPIC ARM OF THE SOCIETY FOR SURGERY OF THE ALIMENTARY TRACT. THE FOUNDATION SUPPORTS THE RESEARCH AND EDUCATION MISSION OF THE SOCIETY. THE HISTORY OF THE SOCIETY DEMONSTRATES THAT RESEARCH IS THE BASIS FOR NEW TECHNIQUES, NEW PROCEDURES, AND ULTIMATELY IMPROVED PATIENT CARE. EDUCATION SUPPORTS THE RESEARCH COMPONENT BY ASSURING THAT THESE NEW DEVELOPMENTS IN SURGERY RAPIDLY BECOME PART OF EVERYDAY PRACTICE FOR THE SURGEONS.

TO FIND A CURE FOR AMYOTROPHIC LATERAL SCLEROSIS (ALS) THROUGH FUNDING RESEARCH.

TO PROVIDE EASIER ACCESS FOR SENECA NATION MEMBERS TO KIDNEY SCREENINGS, DIALYSIS TREATMENT,ADOLESCENT TESTING, THE DEVELOPMENT OF EARLY DETECTION PROCEDURES

Our mission is to find the cause(s) of AHC, develop effective treatments and ultimately a cure, while providing support to the families and children with AHC by funding research to accomplish these goals. Through research, education and family support we have One Mission - END AHC.