CAH is US, CAH is a rare genetic disorder and the endocrine team at Great Ormond Street Hospital appreciate that patients and parents often feel alone in their journey in dealing with this complex disorder. To help and offer support as well as give both parents and older patients the chance to meet, exchange ideas and share experiences, our group CAH IS US was founded. Follow CAH is US on Facebook.
Canadian Addison Society The number of people with Addison’s Disease (Adrenal Insufficiency) varies with surveys in different countries depending on how these surveys are done, but in the United States, between 6 and 11 out of every 100,000 people will be diagnosed with this disease. The statistics are probably the same in Canada.
With this low prevalence of the disease, you may not have met anyone else who has this problem and it can be supportive to discuss your experience with others who have a similar problem.
We hope to help educate not only the medical society but also the public to aspects of the disease.
ADL Life, Adrenoleukodystrophy (ALD) is a rare, inherited metabolic disorder. In this disorder, the fatty covering (myelin sheath) of nerve fibers in the brain is lost, and the adrenal gland degenerates, usually leading to progressive neurological disability and death. A good portrayal of the effects of ALD on both the victim and family is the film Lorenzo’s Oil. It accurately describes the deterioration of a young boy with the disorder and resultant problems. The film does suggest that a cure (Lorenzo’s Oil) was found. Unfortunately, this is not the case for those already with symptoms, although the oil is now a recognised and hopefully preventative treatment for all boys under six with the gene who are not presenting symptoms. It is now also being tested as a preventative treatment for men with adrenomyeloneuropathy (AMN) and females with symptoms. Follow ADL Life Twitter. Read about the link between ALD and AI.
EndocrineWeb has a long legacy as the leading website serving patients and doctors with focused coverage of endocrine disorders and more specifically Thyroid conditions. Link to their pages on Hyperthyroidism Hypothyroidism Thyroid Cancer
Global Genes , Global Genes™ is one of the leading rare disease patient advocacy organizations in the world. The non-profit organization promotes the needs of the rare disease community under a unifying symbol of hope – the Blue Denim Genes Ribbon™. What began as a grassroots movement in 2009 , with just a few rare disease parent advocates and foundations , has since grown to over 500 global organizations.
RDLA, Rare Disease Legislative Advocates (RDLA) is a collaborative organization designed to support the advocacy of all rare disease groups. Our goal is to empower the patient to become an advocate! By growing the patient advocacy community & working collectively we can amplify our many voices to ensure rare disease patients are heard in State & Federal Government.
Team Impact, Team IMPACT matches children with college teams to create an opportunity for children to participate as a team member. Basic eligibility requirements include that the child Is between the ages of 5-15 Has been diagnosed with a life-threatening or chronic illness that has resulted in treatment and/or extended hospital stays within the past 3 years. As a result of their diagnosis, could benefit from a boost to improve their quality of life both socially and psychologically.
harmony 4 hope, Harmony 4 Hope is a not for profit 501c3 tax exempt corporation founded in 2014 for the purpose of using Music to uplift children, create community awareness for rare disease and contribute to Rare Disease Scientific Research.
Pituitary Network Association To support, pursue, encourage, promote and where possible, fund research on pituitary disorders in a sustained and full-time effort to find a cure for these illnesses. The PNA will disseminate information helpful to the medical community, the public and to pituitary patients and their families on matters regarding early detection, symptoms, treatments and resources available to patients with pituitary disease. Watch our Public Service Announcement on YouTube
NORD Every day in America, 30 million people wake up to fight the battle with a rare disease. The vast majority are children. For most, there are no cures and few, if any, proven and effective treatments. NORD provides a unified voice for those courageous individuals, and the parents and other caregivers seeking to help them, so that they won’t have to fight that battle alone.
The Mighty We publish real stories by real people facing real challenges. We are building a brand and a community around them. Having a disability or disease doesn’t have to be isolating. That’s why The Mighty exists.
WEP Clinical is a clinical services company which sets up and manages Expanded Access Programs (EAPs) on behalf of sponsors. These programs, also known as Compassionate Use or Named Patient Programs, distribute potentially life-saving treatments to where there is an unmet medical need. Patients who have no viable treatment options available to them, who cannot receive an investigational drug through a clinical trial, or who live in a country where an approved drug is not commercially available, can potentially gain access to medication through one of these programs.