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How to buy Spinraza (nusinersen): You can order Spinraza (nusinersen) from TheSocialMedwork if the drug has not been approved or is not available in your country. We help patients and doctors get access to medicines at the lowest price worldwide.
For this product the estimated delivery time is usually between 12 and 20 working days.
Spinal Muscular Atrophy
Marketing Authorisation Holder
Biogen Idec Ltd
Mode of Action
Antisense oligonucleotide (ASO)
Regulatory agency approval
European Medical Agency (EMA), European Union, Food and Drug Administration (FDA), USA, Therapeutic Goods Administration (TGA), Australia
Spinraza (nusinersen) is indicated for the treatment of pediatric and adult patients with spinal muscular atrophy (SMA)1,4.
How does Spinraza (nusinersen) work?
SMA is a rare and often fatal hereditary (genetic) disease that causes weakness and muscle wasting due to the loss of motor neurons controlling movement2.
Spinraza (nusinersen) is an antisense oligonucleotide (ASO) that is designed to treat SMA caused by mutations in the chromosome 5q. This mutation leads to the deficiency of a protein, the survival motor neuron (SMN) protein. Spinraza (nusinersen) alters the synthesis of the deficient protein in order to increase production of full-length SMN protein and thereby promoting the maintenance of motor neurons3.
Is Spinraza (nusinersen) approved?
Spinraza (nusinersen) was approved for the treatment of children and adults with spinal muscular atrophy (SMA) by:
FDA (USA) on December 23, 20162
EMA (EU) on May 30, 20175
How do I take Spinraza (nusinersen)?
Spinraza is an injection administered into the fluid surrounding the spinal cord (intrathecal injection)2,5. The standard dosage is:
12 mg / 5 mL per administration
Initiate treatment with 4 loading doses. The first dose should be followed by 3 more doses after 2, 4, and 9 weeks and then one dose every 4 months thereafter. Treatment should be continued for as long as the patient benefits from it1,5.
Complete information about Spinraza (nusinersen) dosage and administration can be found in the resources section.
Consult your treating doctor for personalised dosing.
Common side effects of Spinraza (nusinersen)
Side effects include4:
For a comprehensive list of side effects and adverse reactions please refer to the official prescribing information1,4.
The efficacy of nusinersen for SMA was assessed in a clinical trial (CS3B - ENDEAR) involving 121 patients with infantile-onset SMA who were diagnosed before 6 months of age and who were less than 7 months old at the time of their first dose1,4. Patients were randomised to receive an injection of Spinraza (nusinersen) into the fluid surrounding the spinal cord, or undergo a mock procedure (sham-control) without drug injection (a skin prick)1,4.
At the final analysis, the percentage of responders (patients achieving the pre-defined motor milestone responder criteria) among the nusinersen group was 51% vs 0% among the patients in the control group. Fewer patients died or received permanent ventilation among the nusinersen treated patients (39%) compared with the control group (68%)4. The portion of patients that achieved improvement in total milestone score (which included the ability to kick, head control, rolling, sitting, crawling, standing or walking) was 67% in the patients treated with nusinersen vs 17% in the sham-control group, while the portion of patients that worsened in the two groups was 1% and 22% respectively4. When measured with the Children’s Hospital of Philadelphia Infant Test for Neuromuscular Disease (CHOP INTEND) score, the proportion of patients achieving a 4-point improvement was 73% with nusinersen and 3 % without, while the proportion of those worsening was 7% with nusinersen and 49% without4.
Positive results were achieved also in a study on patients with later onset SMA (after 6 months of age). The study CS4 (CHERISH) involved 126 patients randomised 2:1 to either nusinersen or sham-control. The main measure considered was the Hammersmith Functional Motor Scale Expanded (HFMSE). Among the patients treated with nusinersen, 57.3% achieved an improvement from baseline of at least 3 points vs. 20.5% of the patients in the control group4.
During the transaction process I was very well informed by the TheSocialMedwork team. They have put as much of their effort to offer best service to me. Moreover the medicine I have ordered was imported two weeks earlier then it was expected. I highly recommend The Social Med Work.
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For prescription medicines you will always need a prescription from your treating doctor. This is to ensure that your treatment is monitored and led by a doctor who is responsible for administering the medicine to you. The prescription is the evidence that your doctor gives you their medical support.
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