Muscular Dystrophy Association (MDA) is an American nonprofit organization dedicated to supporting people living with muscular dystrophy, ALS, and related neuromuscular diseases. Founded in 1950 by Paul Cohen, who lived with muscular dystrophy, MDA accelerates research, advances care, and works to empower families to live longer and more independent lives. Renowned for The MDA Labor Day Telethon, the annual telecast aired live from 1966 to 2010 and was hosted by Jerry Lewis, who also served as MDA's national chairman.
Friedreich's ataxia is an autosomal-recessive genetic disease that causes difficulty walking, a loss of coordination in the arms and legs, and impaired speech that worsens over time. Symptoms generally start between 5 and 20 years of age. Many develop hypertrophic cardiomyopathy and require a mobility aid such as a cane, walker, or wheelchair in their teens. As the disease progresses, some affected people lose their sight and hearing. Other complications may include scoliosis and diabetes mellitus.
Idebenone is a drug that was initially developed by Takeda Pharmaceutical Company for the treatment of Alzheimer's disease and other cognitive defects. This has been met with limited success. The Swiss company Santhera Pharmaceuticals has started to investigate it for the treatment of neuromuscular diseases. In 2010, early clinical trials for the treatment of Friedreich's ataxia and Duchenne muscular dystrophy have been completed. As of December 2013 the drug is not approved for these indications in North America or Europe. It is approved by the European Medicines Agency (EMA) for use in Leber's hereditary optic neuropathy (LHON) and was designated an orphan drug in 2007.
The American Diabetes Association (ADA) is a United States-based nonprofit that seeks to educate the public about diabetes and to help those affected by it through funding research to manage, cure and prevent diabetes. It is a network of 565,000 volunteers which includes 20,000 healthcare professionals and administration staff members.
Spinocerebellar ataxia (SCA) is a progressive, degenerative, genetic disease with multiple types, each of which could be considered a neurological condition in its own right. An estimated 150,000 people in the United States have a diagnosis of spinocerebellar ataxia at any given time. SCA is hereditary, progressive, degenerative, and often fatal. There is no known effective treatment or cure. SCA can affect anyone of any age. The disease is caused by either a recessive or dominant gene. In many cases people are not aware that they carry a relevant gene until they have children who begin to show signs of having the disorder.
Susan G. Komen is a breast cancer organization in the United States.
Charity Navigator is a charity assessment organization that evaluates hundreds of thousands of charitable organizations based in the United States, operating as a free 501(c)(3) organization. It provides insights into a nonprofit's financial stability, adherence to best practices for both accountability and transparency, and results reporting. It is the largest and most-utilized evaluator of charities in the United States. It does not accept any advertising or donations from the organizations it evaluates.
The National Psoriasis Foundation (NPF) is the world's largest nonprofit organization serving people with psoriasis and psoriatic arthritis. The NPF provides information and services to help people manage their condition while supporting research to find a cure. In addition to serving more than 3 million people annually through patient and professional health education and advocacy initiatives, the NPF has funded more than $10 million in psoriatic disease research grants and fellowships.
The Lupus Foundation of America (LFA), founded in 1977, is a national voluntary health organization based in Washington, D.C., with a network of chapters, offices and support groups located in communities throughout the United States. The Foundation is devoted to solving the mystery of lupus. Its mission is to improve the quality of life for all people affected by lupus through programs of research, education, support and advocacy.
Colorectal Cancer Alliance is the largest and oldest colorectal cancer non-profit organization in the US.
Breast Cancer Awareness Month (BCAM), also referred to in the United States as National Breast Cancer Awareness Month (NBCAM), is an annual international health campaign organized by major breast cancer charities every October to increase awareness of the disease and raise funds for research into its cause, prevention, diagnosis, treatment, and cure.
Wounded Warrior Project (WWP) is an American charity and veterans service organization that operates as a nonprofit 501(c)(3). WWP offers a variety of programs, services and events for wounded veterans who incurred a physical or mental injury, illnesses, or co-incident to their military service on or after September 11, 2001. Military family members and caregivers are also eligible for WWP programs.
Autism Speaks Inc. is a non-profit autism awareness organization and the largest autism research organization in the United States. It sponsors autism research and conducts awareness and outreach activities aimed at families, governments, and the public. It was founded in February 2005 by Bob Wright and his wife Suzanne, a year after their grandson Christian was diagnosed with autism. The same year as its founding, the organization merged with Autism Coalition for Research and Education. It then merged with the National Alliance for Autism Research in 2006 and Cure Autism Now in 2007.
The Pancreatic Cancer Action Network (PanCAN) is a United States-based 501(c)(3) charity that funds research, provides patient/caregiver support, conducts community outreach and advocates for increased federal research funding for those affected by pancreatic cancer.
BrightFocus Foundation is a nonprofit organization based in Clarksburg, Maryland. BrightFocus funds groundbreaking research in an urgent effort to discover cures for Alzheimer's disease, macular degeneration and glaucoma, and provides expert information and free English and Spanish resources to increase awareness about these diseases. Through its flagship research programs—Alzheimer’s Disease Research, National Glaucoma Research, and Macular Degeneration Research—the Foundation is currently supporting a $75 million portfolio of 287 scientific projects worldwide. BrightFocus has awarded nearly $275 million in groundbreaking medical research funding since inception and supports research and provides public education and free printed brochures and publications on brain and eye diseases, including Alzheimer's disease, macular degeneration and glaucoma. BrightFocus has active research grants in 17 countries and at 154 institutions. BrightFocus Foundation offers free low vision audio chats monthly.
Ovarian Cancer Research Alliance (OCRA) is a not-for-profit organization focused on ovarian cancer research, advocacy and patient support. The organization was formed in January 2016 when the former not-for-profit organization Ovarian Cancer Research Fund, which focused primarily on ovarian cancer research, combined with Ovarian Cancer National Alliance, which focused primarily on ovarian cancer advocacy and support programs, to form one organization.
Cure Alzheimer's Fund (CAF) is an American non-profit organization based in Wellesley, Massachusetts. It supports and funds research focusing on understanding and potentially curing Alzheimer's disease utilizing a venture philanthropy approach, targeting research with the highest probability of preventing, slowing or reversing Alzheimer's disease. The board of directors covers all operating costs so that all donations go entirely to research.
Omaveloxolone, sold under the brand name Skyclarys, is a medication used for the treatment of Friedreich's ataxia. It is taken by mouth.
Kyle Bryant is an athlete, speaker and the spokesperson for the Friedreich's Ataxia Research Alliance (FARA). Bryant is the founder and event director of rideATAXIA – a nationwide bike ride fundraiser benefiting FARA.
Marguerite Virginia Evans-Galea is the co-founder of Women in STEMM Australia. STEMM. Her research is focused on gene therapy and neurodegenerative diseases.
