What are the treatments for Fabry disease?
There is no cure for Fabry disease. Recombinant alpha-galactosidase A (alpha-Gal A), the enzyme that is deficient in patients with Fabry disease, and migalastat hydrochloride, an oral pharmacologic chaperone that facilitates trafficking of alpha-Gal A to lysosomes, are therapeutic options for eligible individuals.
Could Fabry disease have been prevented?
Can Fabry’s disease be prevented? Fabry’s disease is inherited, so it cannot be prevented. However, early diagnosis and an experienced, multidisciplinary medical team can provide the necessary care to slow down the progression of the disease and improve the quality of life in patients suffering from Fabry’s disease.
What is enzyme replacement therapy for Fabry disease?
Enzyme replacement therapy with Fabrazyme restores levels of alpha-galactosidase A. This allows your body to break down lipids. As a result, it can provide relief from Fabry disease symptoms, as well as helps prevent complications.
Is Fabrys disease fatal?
Fabry disease is a rare, inherited disease caused by the deficiency of an enzyme. It is a lipid storage disorder that mainly affects males. The disease can cause long-term difficulties in the kidneys, heart, and nervous system. It can be fatal.
Can Fabry cause hair loss?
Additional symptoms include chronic fatigue, dizziness, headache, generalized weakness, nausea, vomiting, delayed puberty, lack of or sparse hair growth, and rarely malformation of the joints of the fingers.
How long does Fabrazyme infusion take?
Your Fabrazyme infusion time may be approximately 4 to 5 hours or more when you first start treatment, depending on the infusion site’s policies.
What is the treatment for Fabry disease?
Fabry Disease Treatment. In the United States and many other countries there is a commercially approved Enzyme Replacement Therapy (ERT) to treat Fabry disease called agalsidase beta (Fabrazyme®) produced by Sanofi-Genzyme. It is administered by intravenous infusion usually every two weeks for life or until another suitable medication becomes…
What is Fabry disease?
Fabry disease is one of a group of conditions known as lysosomal storage diseases. The genetic mutation that causes Fabry disease interferes with the function of an enzyme that processes biomolecules known as sphingolipids, leading to these substances building up in the walls of blood vessels and other organs.
Can mRNA therapy be used to treat Fabry disease?
Systemic mRNA Therapy for the Treatment of Fabry Disease: Preclinical Studies in Wild-Type Mice, Fabry Mouse Model, and Wild-Type Non-human Primates
Does recombinant alpha-galactosidase a therapy improve mortality in Fabry disease?
Recombinant alpha-galactosidase A (alpha-Gal A), the enzyme that is deficient in patients with Fabry disease, and migalastat hydrochloride, an oral pharmacologic chaperone that facilitates trafficking of alpha-Gal A to lysosomes, are therapeutic options for eligible individuals. The impact of these therapies on mortality is unknown.