Your Health: 3-28-13 Fabry disease - ABC 33/40 - Birmingham News, Weather, Sports

Your Health: 3-28-13 Fabry disease

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     Kidney disease is all too common.   However, there's a rare kidney-related disease that leaves family members questioning whether they will live to see another day.

An Alabaster man was the first patient in the U.S. to received a new drug to treat an enzyme disorder that disable his childhood, attacked his health and his future.

@DOUG MORGAN REMEMBERS A DIS-EASE AND PAIN IN HIS BODY, SINCE CHILDHOOD.

HE TOOK BLOOD PRESSURE MEDICINE WHEN HE WAS IN ELEMENTARY SCHOOL.

Doug Morgan says, "I couldn't play outside with my friends cause I don't perspire I get really hot, overheated turn red and it would cause a lot of pain. The pain was like heightened sensitivity of the skin. A tingling. A burning pain mainly in my feet and hands.

 DOCTORS THEN THOUGHT HE HAD A KIDNEY DISEASE AND RHEUMATIC FEVER.

MORGAN SUFFERED WITH DOULBLE-VISION, DURING COLLEGE...

Morgan says, "You're not able to concentrate , you're dizziness, you can't watch TV, can't really sleep."

...THAT FOLLOWED BY A STROKE.

LATER, DOCTORS DIAGNOSED MORGAN WITH A RARE , UNUSUAL ENZYME DISORDER THAT DESTROYS VITAL ORGANS CALLED FABRY DISEASE.

 UAB Hilda B. Anderson Professor of Nephrology, Dr. David Warnock, M.D. says, "Fabry disease is a rare basic metabolic, inherited disease. It's is transmitted on the female chromosome, on the X chromosome and it involves the heart, brain, the kidneys the skin the GI system."

A LACK OF THE ENZYME CALLED A-GALACTOSIDASE REDUCES THE BREAKDOWN OF A FATTY SUBSTANCE CALLED LIPIDS WHICH BUILD UP TO HARMFUL LEVELS IN THE EYES, KIDNEYS , NERVOUS SYSTEM AND CARDIOVASCULAR SYSTEM.

 Dr. David Warnock says, "The classic history was progressive loss of kidney function to the point that by the age of forty, the patients would require dialysis or transplantation or would die if those were not available."

MORGAN FACED A DIFFICULT TIME.

AT AGE 24, HE WAS DOUBTFUL, HE'D LIVE TO SEE ANOTHER BIRTHDAY.

 Morgan says, "When I had the stroke I was not gonna give up so I may have given up before but not now."

 MORGAN, NOW A CHAPLAIN AT PRINCETON BAPTIST HOSPITAL, HAD FOUND A FRESH FAITH IN A HIGHER POWER.

THE UAB BIOLOGY MAJOR VOLUNTEERED TO TAKE AN EXPERIMENTAL DRUG FOR FABRY DISEASE IN CLINICAL TRIALS IN BETHESDA, MARYLAND.

Morgan says, "I was flying to NIH to get the repligal infusion they were working on the same type drug it's just lower dose."

 THE LOWER-DOSE TEST DRUG DIDN'T STOP MORGAN'S KIDNEY DECLINE AFTER ALMOST TWO YEARS.

 BUT, A STRONGER DRUG WAS IN THE WORKS TO REPLACE THE ENZYME MORGAN WAS MISSING.

 AT AGE 41 MORGAN BECAME THE FIRST U.S. PATIENT TO RECEIVE A FDA-APPROVED INTRAVENOUS INFUSION MEDICATION CALLED FABRAZYME AT UAB IN 2003.

 Dr. Warnock says, "We've been able to stop the loss of kidney function and believe it actually decreases the cardiac involvement and maybe decreasing the ultimate brain involvement in terms of strokes and memory loss."

 BEFORE TAKING THE FABRAZYME INFUSIONS, MORGAN SAYS, HE WASN'T ABLE TO DO MANY FAMILY-FUN ACTIVITIES WITH HIS DAUGHTERS AND WIFE.

BUT, THE DRUG REVIVED HIM, SO TO SPEAK.

Morgan says, " After I was given the Fabrazyme it felt like something had washed through my kidneys to clean them out and I felt much more energetic and able to deal with things."

IT'S BEEN TEN YEARS.   MORGAN CONTINUES TO RECEIVE THE INFUSION TREATMENT.. NOW AT HOME.

Morgan says, "With a needle in the vein, it takes about two hours for me."

MORGAN NEEDS AN INFUSION OF ALMOST TWO VIALS OF FRABRAZYME EVERY TWO WEEKS.

He says, "Sometimes I have a slight reaction because my body sees it as foreign.  It's amazing. It's almost like going to bed with the flu and I wake up and I'm a new person the next day."

AT AGE 51, DOUG MORGAN IS THANKFUL TO BE ALIVE AND ABLE TO FUNCTION AT A HIGH LEVEL.

HOWEVER, HE'S STILL HOPEFUL FOR FURTHER ADVANCES IN ENZYME DISORDER TREATMENT.

 Morgan says, "My hope is they will get away from the infusions I hope they are somewhat uncomfortable..takes a little bit of time.  I'm just hoping that there will be other treatments that may be affective for other people like my children."

 New research is being conducted including aiming to reduce the impact of Fabry disease on the brain and the heart.

Doug Morgan's two daughters are carriers.  One is affected and gets the enzyme infusions every two weeks.  He says, the other daughter shows no signs so far.

Doug's brother also battles ''Fabry'' disease. It's challenged the brother's heart; while kidney function is Doug's greater concern.

Males react differently than females because of the affected X chromosome.

Dr. Warnock says 60 to 70 people in Alabama face an enzyme deficiency.

About half of those patients depend on replacement therapy.

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