Marathon was founded to develop medicines
for rare neurological and movement disorders.
We have deep expertise in earning FDA approvals
and getting effective new medicines to
the patients who need them.
Marathon was founded to develop treatments for rare neurological and movement disorders. Our focus is on treating rare or “orphan” diseases that affect fewer than 200,000 patients each year.1 We have deep expertise in earning FDA approvals for orphan medications and delivering effective medicines to the patients who need them.
There are 6,000 rare diseases that currently have no treatment available. As many as 30 million Americans — nearly one in 10 — have a rare disease.2
Many rare diseases are genetic. According to the National Organization for Rare Disorders, children make up more than half of the people known to be affected by rare diseases.
Marathon Pharmaceuticals is committed to inspiring hope by developing new medicines to treat devastating rare diseases.
Rare diseases are tough to diagnose and even harder to treat. Because they affect small numbers of patients, people living with rare diseases often struggle to obtain a correct diagnosis and most physicians have little or no experience treating rare diseases. The challenge of rare diseases is compounded by a lack of data that could help prevent, diagnose or treat these conditions.
Even though the individual diseases affect a small population, together rare diseases have a significant impact on public health in the U.S. and worldwide.
- “Orphan Drug Act: 21 CFR Part 316.” FDA Website. http://www.fda.gov/ForIndustry/DevelopingProductsforRareDiseasesConditions/ucm2005525.htm. Accessed September 23, 2015.
- “Frequently Asked Questions”. The National Organization for Rare Disorders (NORD) Website. http://rarediseaseday.us/wp-content/uploads/2011/11/RDD-FAQ-2013.pdf. Accessed September 23, 2015.
Duchenne muscular dystrophy
Information and resources on Duchenne Muscular Dystrophy
Research and development
Developing new treatments for rare diseases
Developing new treatments
for people with rare diseases