Judson's Legacy

Treatment for Krabbe Disease

TREATMENT  FOR KRABBE

& DISEASE-MANAGEMENT

 

Treatment for Krabbe Before the Onset of Symptoms

Stem Cell Transplantation (HSCT) has been shown to significantly extend life and improve the quality of life for children with Krabbe Leukodsytrophy; it is currently our most effective treatment for Krabbe. Doctors transfuse healthy, unrelated donor cells with normal GALC enzyme activity, into the Krabbe patient. A successful stem cell transplant stimulates normal development of myelin and reduces neurological symptoms.

However, the key for a successful transplant is that it be performed before a child is symptomatic; once the myelin begins to degrade it is irreparable (though myelin repair is a significant focus of current research).  Therefore, newborn screening is essential for diagnosing Krabbe disease before the onset of symptoms so families have the option for stem cell transplantation as treatment.

Small 5 Dots

Disease-Management After the Onset of Symptoms

Cord Blood

After a child becomes symptomatic, treatment consists of managing symptoms to make the child comfortable as the disease takes its course.  Disease-management often includes:

  • Anticonvulsant medications to deal with seizures
  • Drugs to ease muscle spasticity and irritability
  • Drugs that reduce the incidence of reflux
  • Various therapies, including physical and occupational therapy, to minimize the deterioration of abilities and muscle tone.

 

Judson’s Legacy is committed to funding research so one day all families who are given the news of Krabbe disease have the hope of life for their child.  Please help us make a difference.

 

Newborn Screening      Krabbe Research 

Erik Haines

Meet Erik

Erik has Krabbe disease…BUT…he just graduated from college, having studied Information Technology. He was a competitive swimmer in high school.  Erik was tested for Krabbe after his brother Adam became symptomatic and was diagnosed in 1994 with the disease.  Erik, not yet symptomatic, was able to receive a cord blood transplant that year. Adam, Erik’s brother, was too symptomatic to receive a transplant. Adam died of Krabbe disease just a year after diagnosis.

Two brothers with Krabbe disease. Erik lives and leads a beautiful life. Adam died. Erik was able to receive life-saving treatment because of the life of his brother Adam.

With newborn screening, lives can be saved. Newborn screening allows us to diagnose Krabbe disease before the onset of symptoms, making treatment, like Erik’s, possible. Help us advocate for newborn screening so that families do not have to lose a child to save a child!

 

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