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Reye's Syndrome Awareness

Aspirin should not be given to children

A serious, often fatal disorder

Research has shown a link between Reye's syndrome and the use of aspirin to treat flu-like illnesses and chicken pox in children.

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Family Ties

Genetic testing sounds so clinical, but nothing could be more personal. There are several reasons why you might consider it, one of them being to determine whether or not you or your child runs the risk of developing a certain disease.

More About Your Make-up

Rare Diseases Blog

Girl with "mermaid syndrome" dies at age 10

Wednesday October 28, 2009

Shiloh Pepin, who was born with sirenomelia, or "mermaid syndrome," has died at the age of 10. She passed away last Friday in Maine.

Sirenomelia is a condition in which one's legs are fused together from the waist down. Pepin did not have any genital organs or a large intestine. Most children born with sirenomelia die soon after birth.

You can read more about Shiloh at ABCNews.com.

Folotyn Gets OK for Rare Lymphoma Treatment

Monday September 28, 2009

Though the news might not make a splash for most, those with relapsed and refractory peripheral T-cell lymphoma are rejoicing.The FDA has approved Folotyn (pralatrexate) for for treatment of this rare cancer -- the first treatment ever available.  Toxicity is a concern, and so those being treated with Folotyn are recommended to take folic acid and vitamin B12 supplements.

FDA News Release



Sabril approved by FDA to treat infantile spasms

Tuesday September 1, 2009

On August 21, 2009, the U.S. Food and Drug Administration (FDA) approved Sabril (vigabatrin) to treat infantile spasms in children ages 1 month to 2 years. Infantile spasms are a type of seizure disorder, often part of West syndrome, in which the infant suddenly bends forward at the waist and the body, arms, and legs stiffen. These spasms last a few seconds and occur in clusters of anywhere from 2 to 100 spasms at a time. Some infants have dozens of these clusters of spasms in a day.

Sabril is the first drug in the United States approved to treat infantile spasms. Damage to vision is an important safety concern with the use of Sabril. The drug will have a boxed warning to alert health care professionals to this risk of a progressive loss of peripheral vision with potential decrease in visual acuity. The risk of vision damage may increase based on the dosage and duration of use, but even the lowest doses of Sabril can cause vision damage. Periodic vision testing is required for those taking Sabril. Because of the risk of permanent vision damage, the drug will be available only through a restricted distribution program.

Osteogenesis Imperfecta

Sunday August 30, 2009

Broken arm bonesOsteogenesis imperfecta, also known as brittle bone disease, is an inherited condition. Almost all individuals with osteogenesis imperfecta have fragile bones that break easily.

Photo © A.D.A.M.

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