The CME network for orphan diseases

Areas of Focus

Cystic Fibrosis

Cystic fibrosis (CF) is an inherited chronic disease that affects the lungs and digestive system of about 30,000 children and adults in the United States (70,000 worldwide). A defective gene causes the body to produce unusually thick, sticky mucus that clogs the lungs and leads to life-threatening lung infections. It also obstructs the pancreas and stops natural enzymes from helping the body break down and absorb food.

In the 1950s, few children with cystic fibrosis lived to attend elementary school. Today, advances in research and medical treatments have further enhanced and extended life for children and adults with CF. Many people with the disease can now expect to live into their 30s, 40s and beyond.

Pediatric MS

Childhood Multiple Sclerosis (MS) is not common: Because of the relatively low incidence, pediatricians and most neurologists are not prepared for the specific challenges in the diagnosis and management of the disease in people under 18.

Pediatricians may not be familiar with MS because they are not expecting to see it in children. Other childhood disorders with similar symptoms can make diagnosis of MS in children particularly challenging. An estimated 8,000-10,000 children have multiple sclerosis in the United States, and another 10,000- 15,000 experience disorders that may be related to MS, classifying pediatric MS and its associated disorders as an FDA designated orphan disease.

Pediatric Oncology

While treatment for childhood cancer has improved drastically over the past four decades years from a less than 20 percent survival rate to an approximate 75 percent overall five year cure rate, sadly childhood cancer remains the number one disease killer of our nation's children.

Children face aggressive treatments leaving a trail of devastation for their families. The diagnosis of a child with cancer is often at a time when the family is young and has a limited income. The impact of having a child diagnosed with cancer is deeply felt financially, socially, and emotionally by the entire family.