Spinraza is an innovative treatment for spinal muscular atrophy (SMA), an inherited disorder that affects the motor neurons in the spinal cord, leading to muscle weakness and even paralysis in some cases. But who is responsible for the development of this groundbreaking treatment? In this article, we will explore the journey of Spinraza from research to development and learn more about the people and companies behind this revolutionary therapy.
Spinraza is a medication used to treat spinal muscular atrophy (SMA). It is manufactured and distributed by Biogen, a biopharmaceutical company based in Cambridge, Massachusetts. The drug was approved by the U.S. Food and Drug Administration (FDA) in December 2016. It is given as an injection into the spinal canal. Spinraza is the first approved treatment for SMA and is the only medication available to treat the disease. It has been shown to improve motor function, reduce the risk of scoliosis, and prolong survival in individuals with SMA.
Who Makes Spinraza?
Spinraza is a drug used to treat spinal muscular atrophy (SMA). It is the first and only FDA-approved treatment for this genetic disorder. Spinraza is made by the biotechnology company Biogen.
Biogen
Biogen is a global biotechnology company. They are headquartered in Cambridge, Massachusetts and have operations in over 70 countries. Biogen specializes in researching, developing, and manufacturing therapies for the treatment of neurological and neurodegenerative diseases. Spinraza is one of their most successful products.
Features of Spinraza
Spinraza is an antisense oligonucleotide (ASO) that works by increasing the production of a protein called SMN, which is essential for motor neuron health. It is administered directly into the spinal canal through a lumbar puncture. Spinraza is the only FDA-approved treatment for SMA, and it has been shown to improve the motor function of SMA patients.
The Cost of Spinraza
Spinraza is a costly treatment. It is estimated to cost around $125,000 per patient per year, with an initial loading dose of $625,000. The cost of the drug is covered by most insurance plans, though out-of-pocket expenses may still be incurred. In addition, Biogen offers financial assistance for those who cannot afford the cost of Spinraza.
Frequently Asked Questions
1. Who makes Spinraza?
Spinraza is a prescription medicine used to treat spinal muscular atrophy (SMA). It is manufactured by Biogen, a biopharmaceutical company based in Cambridge, Massachusetts. Biogen has been involved in the development of treatments for neurological diseases for over 30 years and is a leader in the field of gene therapy. The company also has a portfolio of approved therapies for other neurological disorders, including multiple sclerosis and Parkinson’s disease. Spinraza is the first and only FDA-approved treatment for SMA, a rare genetic disorder that affects the ability of nerve cells to communicate with muscles. It works by providing a normal copy of the gene responsible for producing the vital protein, SMN, which is deficient in people with SMA.
2. What is the active ingredient in Spinraza?
The active ingredient in Spinraza is nusinersen, a synthetic antisense oligonucleotide (ASO). It works by binding to specific sites on the SMN2 gene, which causes it to produce more of the SMN protein. Nusinersen is administered through intrathecal injection, which is a process in which a needle is inserted into the lower part of the spinal cord to deliver the medication directly to the fluid that surrounds the nerve cells.
3. How is Spinraza administered?
Spinraza is administered through a series of intrathecal injections. The first dose is given as an eight-hour infusion, followed by four additional doses spaced 12 weeks apart. After this, maintenance doses are given every four months. The injection is usually given in the lower part of the spinal cord, near the lumbar region.
4. What does Spinraza treat?
Spinraza is used to treat spinal muscular atrophy (SMA), a rare genetic disorder that affects the ability of nerve cells to communicate with muscles. It works by providing a normal copy of the gene responsible for producing the vital protein, SMN, which is deficient in people with SMA. Spinraza can improve muscle tone, strength and function, as well as reduce the frequency and severity of SMA-related complications.
5. What are the side effects of Spinraza?
The most common side effects of Spinraza include injection site reactions, fever, headache, vomiting, rash, and upper respiratory tract infections. Other more serious side effects include allergic reactions, breathing problems, muscle weakness, and seizures. It is important to talk to your doctor about any side effects you may experience.
6. How long does Spinraza take to work?
The effects of Spinraza may not be noticeable right away. It can take several weeks or months for the medication to start working. In clinical trials, the most significant improvements were seen after 12 weeks of treatment. It is important to continue treatment as directed in order to maximize the benefits of Spinraza.
In conclusion, Spinraza is a revolutionary drug that has changed the lives of those with spinal muscular atrophy. Developed by Biogen and Ionis Pharmaceuticals, Spinraza has enabled those suffering from this debilitating disease to gain the strength and mobility they need to lead a normal life. With this new treatment, those affected can now receive the treatment they need to live life to the fullest.