Onasemnogene is a groundbreaking gene therapy that has revolutionized the treatment of spinal muscular atrophy (SMA), a rare genetic disorder that affects the motor neurons in the spinal cord. This innovative therapy works by delivering a functional copy of the SMN1 gene to replace the defective gene responsible for SMA.

Clinical trials have shown that Onasemnogene can significantly improve motor function and quality of life in patients with SMA. It is administered as a one-time intravenous infusion, making it a convenient and effective treatment option for both children and adults with this debilitating condition.

As with any medication, Onasemnogene may cause side effects in some patients. These can include fever, vomiting, and elevated liver enzymes. It is important to discuss any concerns or potential side effects with your healthcare provider before starting treatment with Onasemnogene.

It is important to note that Onasemnogene is a prescription medication and should only be administered under the supervision of a qualified healthcare professional. It is not recommended for use in patients with a history of allergic reactions to any of the ingredients in the medication.

Overall, Onasemnogene represents a major advancement in the treatment of SMA and offers hope to patients and their families. If you or a loved one has been diagnosed with SMA, talk to your healthcare provider about whether Onasemnogene may be a suitable treatment option for you.