Cystic fibrosis is a genetic disorder that affects various organs, including the lungs and digestive system. Medications are important for managing symptoms, but there is currently no cure for the condition.

Cystic fibrosis causes thickened mucus to form in the lungs and other organs, leading to severe breathing problems. It is a genetic disorder that can pass to children when both biological parents carry the atypical gene relating to it.

There is currently no cure for cystic fibrosis. However, doctors might use several types of medications to manage symptoms. For example, some medications clear the airways of mucus to allow people to breathe.

This article gives an overview of the medications for cystic fibrosis.

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Cystic fibrosis transmembrane conductance regulator (CFTR) modulators are a class of medications that help improve the function of the atypical protein causing cystic fibrosis.

According to the Cystic Fibrosis Foundation (CFF), these drugs work by improving the production, function, or stability of the CFTR protein, helping reduce the symptoms of cystic fibrosis.

However, these medications are only effective in people with specific CFTR mutations. Therefore, doctors will choose the type of CTFR modulator depending on the specific mutation someone has. Some approved CFTR modulators for cystic fibrosis include:

  • a triple combination medication of elexacaftor, tezacaftor, and ivacaftor that could help up to 90%Trusted Source of people with cystic fibrosis
  • ivacaftor
  • a combination of ivacaftor and lumacaftor or tezacaftor

The medications can come in various forms, such as tablets or granules for oral consumption. The specific dosage and types of administration of CFTR modulators will depend on several factors, including other medications and conditions.

Side effects

These medications can cause side effects that may include:

A person needs to contact a doctor immediately if they experience:

Bronchodilators are medications that relax the musclesTrusted Source around the airways to widen them. They allow more air to pass through the lungs, improving breathing. A doctor may prescribe these medications to people with cystic fibrosis before recommending any others.

The CFF notes that examples of bronchodilators include albuterol and levalbuterol hydrochloride (Xopenex). People typically inhale these medications through an aerosol canister. They need to shake the inhaler, squeeze it while breathing the medication in, and hold their breath for a few seconds. Alternatively, some people may use a liquid form of the medication with a nebulizer.

Side effects

Some side effects of bronchodilators include:

In rare cases, bronchodilators can cause serious side effects, such as chest pain, difficulty breathing, and a fast, pounding heartbeat. People who experience these side effects must contact a doctor immediately.

Mucus thinners, or mucolyticsTrusted Source, are a group of medications that reduce the thickness of mucus in the airways. Thinner mucus is easier to clear through coughing or other airway-clearing techniques. People usually inhale mucus thinners, which work well with bronchodilators that widen the airways to make it easier for thinner mucus to exit.

The CFF notes that there are different types of mucus thinners, such as:

  • Hypertonic saline: This medication uses salt to attract water from the mucus and thin it. People typically inhale the medication as a mist twice per day.
  • Mannitol: This powder thins mucus through unknown mechanisms. People typically use the medication with an inhaler in the morning and evening.
  • Dornase alfa: This medication “cuts” DNA strands in white blood cells into shorter pieces to break up mucus. People use nebulizers to inhale the medication once or twice a day.

Side effects

These medications can have various side effects, including:

A person needs to contact a doctor immediately if they experience:

People with cystic fibrosis may require antibiotics to treat or preventTrusted Source lung infections. According to the American Lung Association (ALA), people with cystic fibrosis are at a higher risk of lung and respiratory infections due to the buildup of mucus.

There are many types of antibiotics that someone with cystic fibrosis might receive. People might inhale the medications or take them orally or intravenously (via the veins).

Side effects

Some possible side effects of antibiotics include:

  • nausea and vomiting
  • diarrhea
  • headache
  • temporary tooth discoloration

People need to contact a doctor immediately if they experience:

  • rashes, hives, or other skin problems
  • wheezing
  • itching
  • difficulty swallowing or breathing
  • swelling
  • severe diarrhea that may occur with fever and abdominal cramps
  • prolonged diarrhea, vomiting, fatigue, or paleness within 1 to 4 hours of taking antibiotics such as amoxicillin

Cystic fibrosis is a complex condition that may require several types of treatmentsTrusted Source beyond medication. These can include:

Cystic fibrosis is an incurable genetic condition that causes mucus to build up, leading to breathing problems and other symptoms. Several treatments are available to manage symptoms.

Cystic fibrosis medications include bronchodilators, cystic fibrosis transmembrane conductance regulator (CFTR) modulators, mucus thinners, and antibiotics.

Doctors may also prescribe other treatments with medications, such as oxygen therapy or airway clearance techniques.