What is FA?
FA is a rare, progressive disease that damages the nervous system, causing you to lose control of your muscles over time.
What causes FA?
FA happens when a specific gene, called the frataxin gene (or FXN), does not function properly and cannot help nerve cells work the way they should.
How is FA diagnosed?
FA can be confirmed with a genetic test. It is important that the test include a Friedreich Ataxia Repeat Expansion Analysis.
What happens as FA progresses?
As you get older, FA continues to damage nerve cells, which leads to greater difficulty with things like:
Fine motor skills
How can FA progression be measured?
The severity of FA can be measured in different ways—one way is the modified Friedreich Ataxia Rating Scale (mFARS)
Most people with FA will need to use a wheelchair around 10 to 15 years after they are first diagnosed
Unfortunately, complications from FA can contribute to a shorter average life expectancy
What is SKYCLARYS?
SKYCLARYS is used for the treatment of Friedreich ataxia in adults and children 16 years of age and older.
Before taking SKYCLARYS, tell your healthcare provider about all your medical conditions, including if you:
- have liver problems.
- have a history of heart problems, including heart failure.
- have a high level of fat in your blood (high blood cholesterol).
- are pregnant or plan to become pregnant.
- It is not known if SKYCLARYS will harm your unborn baby.
- Women who use hormonal birth control should use another form of birth control such as a non-hormonal intrauterine system or an extra non-hormonal birth control such as condoms while using SKYCLARYS and for 28 days after stopping SKYCLARYS.
- are breastfeeding or plan to breastfeed. It is not known if SKYCLARYS passes into your breast milk. Talk to your healthcare provider about the best way to feed your baby if you take SKYCLARYS.
Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Some medications interact with SKYCLARYS. Keep a list of your medicines to show to your healthcare provider and pharmacist when you get a new medicine.
What are the possible side effects of SKYCLARYS?
SKYCLARYS may cause serious side effects, including:
- increase in blood liver enzymes. Some people taking SKYCLARYS have had an increase in
the level of liver enzymes in their blood. Your healthcare provider will do liver function tests
- before you start taking SKYCLARYS
- every month for the first 3 months after starting your treatment with SKYCLARYS
- during certain times as needed while taking SKYCLARYS
If your liver enzymes increase, your healthcare provider may change your dose during treatment, stop treatment for some time, or completely stop treatment with SKYCLARYS.
- increase in a blood protein called B-Type Natriuretic Peptide (BNP). BNP tells how
well your heart is
working. Your healthcare provider will check your BNP levels before your treatment with SKYCLARYS. Tell
your healthcare provider if you have signs and symptoms of your heart not working well such as too much
fluid in your body (fluid overload). Signs and symptoms may include:
- sudden weight gain (3 pounds or more of weight gain in 1 day, or 5 pounds or more of weight gain in 1 week)
- swelling in your arms, hands, legs, or feet (peripheral edema)
- fast heartbeat (palpitations)
- shortness of breath
If you have symptoms of fluid overload that is considered a side effect of SKYCLARYS, your healthcare provider may stop treatment with SKYCLARYS.
- changes in cholesterol levels. Increases in low density lipoprotein cholesterol (LDL-C) or bad cholesterol and decreases in high density lipoprotein cholesterol (HDL-C) or good cholesterol have happened during treatment with SKYCLARYS.
Your healthcare provider will check your cholesterol levels before and during your treatment with SKYCLARYS.
The most common side effects of SKYCLARYS include:
- increased liver enzymes (ALT/AST)
- stomach pain
- muscle pain
These are not all the possible side effects of SKYCLARYS. Call your doctor for medical advice about side effects.
You are encouraged to report negative side effects of prescription drugs to the FDA.