Learn how early intervention, proper management strategies, and clinical trials are key for rare disease patients.
There are approximately 7,000 known rare diseases, but only 5% of them have an approved treatment. Learn how early intervention, proper management strategies, and clinical trials are key for rare disease patients.
Friedreich’s Ataxia (FA) is a progressive and debilitating neurological disorder. FA is the most common inherited ataxia and is caused by a mutation on both copies of the FXN gene which makes frataxin protein, which is needed for proper function of the brain, nervous system, and heart.
Clinical trials are an opportunity for patients to receive promising new therapies, but they can be difficult to navigate. Learn how The Leukemia & Lymphoma Society’s Clinical Trial Support Center is helping guide patients.
