TREATMENT FOR KRABBE
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.
Disease-Management After the Onset of Symptoms
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.