New to this website and any advice is appreciated

Hello- My name is Michele. My husband is 44 and  has been diagnosed with Superficial Siderosis. It is a very rare neurodegenerative condition caused by chronic hemosiderin deposition in the brain and spinal cord.  My husband has lost hearing in both ears and now relies on two cochlear implants. As the diesease progresses- the symtoms are similar to Parkinson's Disease with many other debilitating side effects.The disease just progressed and progressed and we hoped and waited. He was fortunate to be able to particiapte in a study using liquid Ferriprox. He had no issues with the medicine and his body responded very well. He had weekly blood tests to be sure his counts were at safe levels and never had any adverse side effects. He was diagnosed 10 years ago and at that time no treatment was available. We were so excited that a medicine was showing improvement for patients with the hemosiderin deposits because for 10 years we and others have had not had any available treatment . Ferriprox is legal and FDA approved here is the United States where we live but not to treat his Superficial Siderosis specifically. Unfortunately since the disease is very uncommon- I am hopeful but  doubtful that we will get FDA aprroval  for Ferriprox to treat Superficial Siderosis specifically. The process is  very long and there are not that many cases. Due to this situation- our insurance has rejected our request for the medicine. It is so upsetting to know something is available that works after waiting 10 years and you cannot get it because it has not been approved for your "specific" disease but has shown to be affective for your disease.

I am not sure how this works in other countries. Is this medicine difficult to purchase? How much does it cost per month? Do we need a prescription? I would appreciate any feedback or input that anyone can share. Has anyone had any adverse side effects with this medicine over a long period of time?

Thanks for taking the time to read this-
Michele

I am making some inquiries to see if I get information about a compassionate use program for Ferriprox. Most drug companies run special programs to help people when there insurance does not cover drug costs.

Hi Andy-
Thanks for checking into this. We mailed  an appeal to the decision today and I am still hoping... The insurance was quick to deny us on the first request so I hope to know something soon either way. Any information that you find would be very much appreciated.
Thanks again,
Michele

Hi Michele,

I contacted Eileen Scott at CAF and she gave me this info,

Hi Andy,
She can contact me or you can just give her the information below, whatever is easier for you.  If he was participating in a study, he might be eligible for the patient assistance program they have set up.
Let me know if you need anything else.
Eileen
 
Ferriprox Total Care at (866) 758-7071
fax (866) 565-7794.
Correspondence can be sent to
Ferriprox Total Care Program
17877 Chesterfield Airport Road,
Chesterfield, Missouri  63005.
 Email is info@centrichealthresources.com
 
 
Eileen Scott
Patient Services Manager
Cooley's Anemia Foundation
330 Seventh Avenue
Suite 200
New York, NY 10001
212-279-8090 ext. 205
212-279-5999

Thanks so much Andy. I am calling her now.
Michele