Name of the Procedure:

Injection, Golodirsen, 10 mg (HCPCS Code J1429)

• Common Name: Golodirsen Injection
• Technical/Medical Term: Golodirsen is also known under the brand name Vyondys 53.

Summary

Injection of Golodirsen is a medical treatment involving the administration of a specific medication to aid in the treatment of specific genetic disorders. The medicine is administered via an injection, usually into a vein.

Purpose

Golodirsen is primarily used to treat Duchenne Muscular Dystrophy (DMD) in patients with a confirmed exon 53 mutation in the dystrophin gene. This procedure aims to increase the production of dystrophin, a protein needed for muscle health.

Indications

• Diagnosed Duchenne Muscular Dystrophy with a confirmed exon 53 mutation.
• Patients who demonstrate progressive muscle weakness and deterioration associated with DMD.
• Physician confirmation that the patient will benefit from exon-skipping therapy.

Preparation

• Pre-procedure Instructions: Patients may need to discuss their current medications and health conditions with their healthcare provider. Fasting is typically not required.
• Diagnostic Tests: Genetic testing to confirm the presence of an exon 53 mutation in the dystrophin gene may be required.

Procedure Description

1. Setup: The patient is seated or lying down, usually at a medical facility.
2. Injection: Golodirsen is administered intravenously (IV) through a needle placed into a vein, typically in the arm.
3. Monitoring: The healthcare provider monitors the patient for any immediate reactions during and after injection.

Tools and Equipment: IV needle, syringe, and Golodirsen medication.

Anesthesia/Sedation: Not typically required, but local anesthesia may be used at the injection site for comfort.

Duration

The injection process usually takes about 30 to 60 minutes.

Setting

The procedure is commonly performed in outpatient clinics, hospitals, or specialized infusion centers.

Personnel

• Registered Nurse (RN) or specialized infusion nurse to administer the injection.
• Physician or specialist overseeing the treatment and patient condition.

Risks and Complications

• Common Risks: Mild pain or discomfort at the injection site, headache, nausea.
• Rare Risks: Severe allergic reactions, kidney toxicity, or respiratory issues.
• Management: Immediate medical attention and medications to manage any adverse reactions.

Benefits

• Potential slowing of muscle deterioration in patients with DMD.
• Improved muscle function and delayed progression of the disease.
• Benefits are typically observed over several months of consistent treatment.

Recovery

• Post-procedure Care: Patients may be monitored for a short time after the injection for any adverse reactions.
• Expected Recovery: No downtime is usually required; patients can resume normal activities shortly after the procedure.
• Follow-up: Regular follow-up appointments to monitor progress and assess the effectiveness of therapy.

Alternatives

• Other Treatment Options: Corticosteroids, other exon-skipping therapies, physical therapy, and supportive care for muscle function.
• Pros and Cons: Corticosteroids can help manage inflammation but may have more side effects. Physical therapy is beneficial but doesn't address the genetic cause of DMD.

Patient Experience

• During: The patient might feel a slight pinch or discomfort from the needle insertion.
• After: Minimal discomfort; normal daily activities can usually be resumed. Pain management is generally not necessary, but over-the-counter pain relief can be used if needed.