Spinraza, it is a beautiful word to SMA families. It's not a cure, but finally a treatment. I'm so glad I was able to see this happen in my lifetime. This means so much to the SMA community. I'll try to post some videos of patients on this drug, and of course links. Do your research, and I hope it will help you.
Unfortunately for us, Declan will not be taking this drug. Since he tested negative for SMA but positive for one mutation with SMARD, this drug will not help him. I'm hoping since we provided a treatment for SMA, one is close for SMARD. May this treatment help the many, but our goal always and forever will be to come up with a cure.
INFO:
- SPINRAZA is a survival motor neuron-2 (SMN2)-directed antisense oligonucleotide indicated for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients (1)
- SPINRAZA is administered intrathecally (Intrathecal administration is a route of administration for drugs via an injection into the spinal canal, or into the subarachnoid space so that it reaches the cerebrospinal fluid (CSF) and is useful in spinal anaesthesia, chemotherapy, or pain management applications.)
- Initiate SPINRAZA treatment with 4 loading doses; the first three loading doses should be administered at 14-day intervals; the 4th loading dose should be administered 30 days after the 3rd dose; a maintenance dose should be administered once every 4 months thereafter.
- SPINRAZA is an antisense oligonucleotide (ASO) designed to treat SMA caused by mutations in chromosome 5q that lead to SMN protein deficiency. Using in vitro assays and studies in transgenic animal models of SMA, SPINRAZA was shown to increase exon 7 inclusion in SMN2 messenger ribonucleic acid (mRNA) transcripts and production of full-length SMN protein. (meaning it replaces a missing protein.)
- Cost: Spinraza, will not come cheap — and, by some estimates, will be among the most expensive drugs in the world. Biogen, which is licensing Spinraza from Ionis Pharmaceuticals, said this week that one dose will have a list price of $125,000. That means the drug will cost $625,000 to $750,000 to cover the five or six doses needed in the first year, and about $375,000 annually after that, to cover the necessary three doses a year. Patients will presumably take Spinraza for the rest of their lives. (I am unable to find at this time what the actual costs are.)
______________________WARNINGS AND PRECAUTIONS ____________________
• Thrombocytopenia and Coagulation Abnormalities: Increased risk for bleeding complications; testing required at baseline and before each dose.
• Renal Toxicity: Quantitative spot urine protein testing required at baseline and prior to each dose.
VIDEOS:
There are many more videos out there about how this treatment is working. I love it!!!
LINKS:
Farrell Family
spinraza
prescribing-information.pdf
curesma
Biogen’s SPINRAZA
https://www.nytimes.com
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