Children's Health Medical Reference

Joenja (leniolisib) is the first treatment approved for APDS. Learn how it works, who it's for, and what studies found.

Learn about possible Fayuvi side effects, how to help manage them, and what to know about vaccines after treatment.

Fayuvi (rebisufligene etisparvovec-hopf) is a one-time gene therapy for Sanfilippo syndrome type A. Here's how it works and what studies found.

Gazyva is now approved to help prevent relapse in children and young adults with nephrotic syndrome. Here's how it works and what to know about the side effects.

Alagille syndrome-related itching can be a challenge for kids. Learn about home remedies that may provide some extra relief.

Every case of Alagille syndrome is different, and your child’s treatment will depend on what symptoms they have. Learn more below.

Get tips on ways to soothe your child's PFIC itch at home — from cool baths to bedtime tricks that help everyone sleep.

There’s no cure for PFIC, but there are treatments available to help better manage symptoms such as itching and potentially slow down liver damage.

Your PFIC care team is invaluable when it comes to management of your child’s condition. Meet the key players, such as a hepatologist (a liver specialist).

PFIC can have far-reaching effects in your child’s body. Learn how the condition may impact digestion, growth, development, and other organ systems.

When your child has Alagille syndrome, their care team will consist of multiple specialists. Here’s what you should know.

This rare genetic condition leads to short stature and sometimes other health concerns. Learn the symptoms, causes, and treatments.

You can’t cure PKU, but medications available today can make life easier for your child with fewer restrictions on their diet. Learn more about your options and their pros and cons.

Learn how a PKU diet protects brain development, which foods to avoid, why medical formula matters, and tips for managing phenylketonuria.

Learn more about PKU, including what the PAH gene does in the body, effects of untreated PKU, and how to manage it effectively to prevent these outcomes.

Kresladi is a gene therapy. Here’s how it treats leukocyte adhesion deficiency type 1 (LAD-1).

Kresladi is a gene therapy for leukocyte adhesion deficiency type 1 (LAD-1). Here’s what to know about its side effects and drug interactions.

Elevidys is a gene therapy treatment. Here’s how it treats Duchenne muscular dystrophy.

Avlayah is a prescription enzyme replacement therapy. Here’s how it treats Hunter Syndrome.

Yuviwel is an FDA-approved medicine to help improve growth rates in certain people with achondroplasia. Here’s how it works.

Redemplo is a prescription injection. Here’s how it treats familial chylomicronemia syndrome (FCS).

Waskyra is a prescription gene therapy. Here’s how it treats Wiskott-Aldrich Syndrome.

Loargys is a prescription enzyme replacement therapy. It is used to treat arginase 1 deficiency (ARG1-D) in adults and children aged 2 or older.

Zycubo is a copper replacement therapy. Here’s how it treats Menkes disease.

Viltepso is a prescription medicine used to treat Duchenne muscular dystrophy (DMD). It is for people who have a specific type of genetic mutation.

Agamree is a once-daily oral medicine. Here’s how it treats Duchenne muscular dystrophy (DMD).

Here’s what you need to know about how vaccines for bacterial meningitis work, what the different types are, and what side effects they could cause.

Here's what you need to know about vaccines and healthy habits that can greatly boost your child's chances of avoiding bacterial meningitis.

As your child gets older, it’s a good idea to make yourself aware of the symptoms of a serious illness called meningitis.

There are different types of meningitis, and one kind in particular poses a risk to teenagers and young adults. Learn about their risk and the things that drive it up.

Ojemda is a targeted cancer medicine. Here’s how it treats low-grade glioma.

Amondys is an antisense oligonucleotide. Here’s how it treats Duchenne muscular dystrophy (DMD)

Ngenla is a long-acting prescription human growth hormone analog. Here’s how it treats growth failure in children with growth hormone deficiency.

Kygevvi is a combination of doxecitine and doxribtimine. Here’s how it treats thymidine kinase 2 deficiency.

Sogroya is a human growth hormone analog. Here’s how it treats growth hormone deficiency.

Achondroplasia can affect your child’s school life. Learn what you can do to help them succeed in the classroom.

It’s important to learn the achondroplasia health issues your child may face. Doing so can help you better support your child as they grow.

Find out what to expect after your child’s achondroplasia diagnosis. Learn about their development and accommodations to help them as they grow.

Pediatric growth hormone deficiency is a rare condition that affects more than your child’s height. Here’s what you need to know.

Learn more about Waardenburg syndrome, a group of inherited genetic disorders that are known for their distinctive physical features and hearing loss.

Pombiliti is a prescription enzyme replacement medicine. Here’s how it treats Pompe disease.

Crenessity is a medicine that regulates adrenal androgens. Here’s how it treats congenital adrenal hyperplasia (CAH).

Opfolda is an enzyme stabilizer. Here's how it treats Pompe disease.

Batten disease is a rare kind of disorder that affects the nervous system. Learn what causes Batten disease and how to identify symptoms.

Speech therapy can improve communication in kids and adults alike. Learn more about what it is, how it works, and what to expect.

Vykat XR is an oral prescription medicine. Here’s how it treats Prader-Willi syndrome.

Learn more about this neurological condition that affects spatial awareness, motor skills, and social cues.

Alyftrek is a combination of three medicines. Here’s how it treats cystic fibrosis.

Tryngolza is an RNA-targeted medicine. Here’s how it lowers triglyceride levels.