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We See You!

Railroad Children is a rare disease consulting firm that works with young patients (ages 0 - 18 years old) and their family, clinicians, and industry to identify novel treatments for even the rarest of diseases. Our mission is to provide medication and treatment solutions to rare diseases with a genetic, congenital, metabolic and infectious origin.  We ensure that the latest advancements in the field are utilized to their full potential, by implementing our deep expertise in molecular biology, genetics, biochemistry and human physiology to identify individualized treatment approaches.  We employ numerous treatment approaches including drug repurposing, identification of novel small molecules, access to investigational medications, and identification of therapies and strategies to improve the quality of life of patients and their family.  Our team of experts is dedicated to providing the highest quality of care to our patients from the point of diagnosis and beyond. We are committed to making a difference in the lives of those affected by rare diseases.

Child in Air Yoga


What do we know about rare diseases?

There are approximately 6000 rare genetic disorders worldwide, and 83% of these disorders affect children. Rare disorders that occur in childhood are particularly devastating because 90% of them compromise the nervous system.  There are three main categories of genetic mutations, 10% of mutations are X-linked, and occur on the sex chromosomes, 32% are autosomal dominant, which means the mutation occurs in only one copy of the same gene, and  45% are autosomal recessive, which means that the mutation is found in both copies of the same gene.  There are different mutations across genetic disorders, which depend on the change to the gene, these mutations include: point mutations, chromosomal mutations, and copy number variants.

At Railroad Children, we have a team of professionals with years of experience in the field of neurology, genetics, molecular biology, biochemistry and human physiology. Our team includes experts in various fields such as genetics, pharmaceuticals, and clinical research.  We were brought together by Sarrita Adams who is the main point of contact for the work we perform.  To learn more about Sarrita click here

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