Eflornithine, sold under the brand name Vaniqa among others, is a medication used to treat African trypanosomiasis (sleeping sickness) and excessive hair growth on the face in women. Specifically it is used for the 2nd stage of sleeping sickness caused by T. b. gambiense and may be used with nifurtimox. It is used by injection or applied to the skin.Common side effects when applied as a cream include rash, redness, and burning. Side effects of the injectable form include bone marrow suppression, vomiting, and seizures. It is unclear if it is safe to use during pregnancy or breastfeeding. It is recommended typically for children over the age of 12.Eflornithine was developed in the 1970s and came into medical use in 1990. It is on the World Health Organizations List of Essential Medicines. In the United States the injectable form can be obtained from the Centers for Disease Control and Prevention. In regions of the world where the disease is common eflornithine is provided for free by the World Health Organization. Medical uses
Sleeping sickness
Sleeping sickness, or trypanosomiasis, is treated with pentamidine or suramin (depending on subspecies of parasite) delivered by intramuscular injection in the first phase of the disease, and with melarsoprol and eflornithine intravenous injection in the second phase of the disease. Efornithine is commonly given in combination with nifurtimox, which reduces the treatment time to 7 days of eflornithine infusions plus 10 days of oral nifurtimox tablets.Eflornithine is also effective in combination with other drugs, such as melarsoprol and nifurtimox.
Mental development, physical development, and height are usually normal. Most patients with Pyles diseases will have symptoms that vary from person to person. Symptoms are:5%–29% of people have

Delated eruption of teethOther people may have

Abnormality of thorax
Absent paranasal sinuses
Arthralgia
Carious teeth
Genu valgum
Hypoplastic frontal sinuses
Limited elbow extension
Mandibular prognathia
Metaphyseal widening
Metaphyseal dysplasia
Muscle weakness
Platyspondyly
Reduced bone mineral density
Scoliosis
Thickened calvaria

Cause
Pyle disease is caused by mutations in the SFRP4 gene. This gene provides instructions for making a protein that blocks (inhibits) a process called Wnt signaling, which is involved in the development of several tissues and organs throughout the body. In particular, regulation of Wnt signaling by the SFRP4 protein is critical for normal bone development and remodeling. Bone remodeling is a normal process in which old bone is broken down and new bone is created to replace it. Mutations in the SFRP4 gene are thought to prevent the production of functional SFRP4 protein. The resulting dysregulation of Wnt signaling leads to the bone abnormalities characteristic of Pyle disease.Pyle disease is inherited in an autosomal recessive pattern, which means both copies of the SFRP4 gene in each cell have mutations. The parents of an individual with an autosomal recessive condition each carry one copy of the mutated gene. While they do not develop the condition, they may have mild abnormalities of the long bones. Diagnosis
There are two clinical molecular genetic test that are available to those thought to have Pyle’s Disease. Theses test are; Sequence analysis of the entire coding region and Deletion/duplication analysis. Differential diagnosis
Pyle disease may be confused with craniometaphyseal dysplasia.