Results for tag: "Adrenoleukodystrophy Drug"

Adrenoleukodystrophy (ALD) is a rare genetic disorder that affects the nervous system and adrenal glands. It is caused by a mutation in the ABCD1 gene, which is responsible for the production of a protein that helps break down very long-chain fatty acids. Symptoms of ALD include progressive neurological deterioration, seizures, and adrenal insufficiency. Treatment for ALD is limited to supportive care and dietary management. The Adrenoleukodystrophy Drug market is a subset of the Central Nervous System Drugs market. It is a small but growing market, with a focus on developing treatments for ALD. Currently, there are no approved drugs for ALD, but several companies are researching potential therapies. These include gene therapy, enzyme replacement therapy, and stem cell therapy. Companies in the Adrenoleukodystrophy Drug market include Sanofi, Pfizer, and Sangamo Therapeutics. Sanofi is researching gene therapy for ALD, while Pfizer is developing an enzyme replacement therapy. Sangamo Therapeutics is researching a stem cell therapy for ALD. Show Less Read more