Description
Golodirsen injection is used to treat Duchenne muscular dystrophy (DMD) in patients with a confirmed specific genetic mutation. DMD is a rare, inherited muscle disease that is caused by absence of a protein called dystrophin, which helps keep muscle cells intact.
This medicine is to be given only by or under the direct supervision of a doctor.
This product is available in the following dosage forms:
- Solution
Before Using
In deciding to use a medicine, the risks of taking the medicine must be weighed against the good it will do. This is a decision you and your doctor will make. For this medicine, the following should be considered:
Allergies
Tell your doctor if you have ever had any unusual or allergic reaction to this medicine or any other medicines. Also tell your health care professional if you have any other types of allergies, such as to foods, dyes, preservatives, or animals. For non-prescription products, read the label or package ingredients carefully.
Pediatric
Appropriate studies performed to date have not demonstrated pediatric-specific problems that would limit the usefulness of golodirsen injection in children.
Geriatric
No information is available on the relationship of age to the effects of golodirsen injection in geriatric patients. DMD is usually a disease of children and young adults.
Breastfeeding
There are no adequate studies in women for determining infant risk when using this medication during breastfeeding. Weigh the potential benefits against the potential risks before taking this medication while breastfeeding.
Drug Interactions
Although certain medicines should not be used together at all, in other cases two different medicines may be used together even if an interaction might occur. In these cases, your doctor may want to change the dose, or other precautions may be necessary. Tell your healthcare professional if you are taking any other prescription or nonprescription (over-the-counter [OTC]) medicine.
Other Interactions
Certain medicines should not be used at or around the time of eating food or eating certain types of food since interactions may occur. Using alcohol or tobacco with certain medicines may also cause interactions to occur. Discuss with your healthcare professional the use of your medicine with food, alcohol, or tobacco.
Other Medical Problems
The presence of other medical problems may affect the use of this medicine. Make sure you tell your doctor if you have any other medical problems, especially:
- Kidney disease—Use with caution. May make this condition worse.
Proper Use
A nurse or other trained health professional will give you or your child this medicine in a hospital. This medicine is given through an IV catheter that is placed in one of your veins. It must be given slowly, so the IV should remain in place for 35 to 60 minutes once a week.
Precautions
Your doctor will check your or your child's progress closely while receiving this medicine to see if it is working properly and to decide if you or your child should continue to receive it. Blood and urine tests may be needed to check for unwanted effects.
This medicine may cause serious allergic reactions, including anaphylaxis, which can be life-threatening and require immediate medical attention. Check with your doctor right away if you have chest tightness, cough, difficulty swallowing, dizziness, fast heartbeat, hives, itching, skin rash, puffiness or swelling around the eyes, face, lips, or tongue, trouble breathing, or unusual tiredness or weakness.
Check with your doctor right away if you or your child have blood in the urine, change in frequency of urination or amount of urine, drowsiness, increased thirst, loss of appetite, nausea or vomiting, swelling of the feet or lower legs, trouble breathing, or weakness. These could be symptoms of a serious kidney problem.
Side Effects
Along with its needed effects, a medicine may cause some unwanted effects. Although not all of these side effects may occur, if they do occur they may need medical attention.
Check with your doctor or nurse immediately if any of the following side effects occur:
More common
- Fast, pounding, or irregular heartbeat or pulse
- fever
Incidence not known
- Agitation
- chest tightness
- coma
- confusion
- cough
- decreased urine output
- depression
- difficulty swallowing
- dizziness
- fast heartbeat
- headache
- hives, itching, skin rash
- hostility
- irritability
- lethargy
- muscle twitching
- nausea
- puffiness or swelling of the eyelids or around the eyes, face, lips, or tongue
- rapid weight gain
- seizures
- stupor
- swelling of the face, ankles, or hands
- trouble breathing
- unusual tiredness or weakness
Some side effects may occur that usually do not need medical attention. These side effects may go away during treatment as your body adjusts to the medicine. Also, your health care professional may be able to tell you about ways to prevent or reduce some of these side effects. Check with your health care professional if any of the following side effects continue or are bothersome or if you have any questions about them:
More common
- Back pain
- bleeding, blistering, burning, coldness, discoloration of the skin, feeling of pressure, hives, infection, inflammation, itching, lumps, numbness, pain, rash, redness, scarring, soreness, stinging, swelling, tenderness, tingling, ulceration, or warmth at the injection site
- constipation
- diarrhea
- falling
- headache
- muscle aches
- sore throat
- stomach pain
- stuffy or runny nose
- vomiting
Other side effects not listed may also occur in some patients. If you notice any other effects, check with your healthcare professional.
Call your doctor for medical advice about side effects. You may report side effects to the FDA at 1-800-FDA-1088.
DRG-20479542
