Marathon's mission is to develop new medicines to treat devastating rare diseases

Rare neurological disorders

Rare neurological conditions can affect how patients’ brains control their bodies. Patients with neurological disorders may notice changes in their ability to walk, talk and care for themselves. The most devastating of these diseases are crippling or fatal.

Nearly 1 in 6 people worldwide suffer from neurological disorders – diseases of the nervous systems influencing everything from cognitive ability to basic muscle control. Many of these disorders are treatable, though the treatment of rare disorders, affecting only a small percentage of the patient population, often remains limited by insufficient data, research, and resources.

As part of Marathon’s commitment to improving patients’ lives, we are developing potential treatments for rare neurological disorders, including:

  • Duchenne Muscular Dystrophy
  • Pediatric Juvenile Idiopathic Arthritis
  • Neuromyelitis Optica (Devic’s Disease)
  • Idiopathic Inflammatory Myopathies
  • Catastrophic Pediatric Epilepsy
  • Multiple Sclerosis

Alongside industry partners and patient advocates, Marathon seeks to acquire promising experimental treatments for rare neurological disorders. Marathon works to bring effective medicines to patients by performing comprehensive clinical trials, obtaining FDA approval, and ensuring efficient delivery for those in need.

Duchenne muscular dystrophy

Information and resources on Duchenne mucsular dystrophy


Research and development

Investing in new rare disease treatments