Children's Health Medical Reference

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.

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

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

Learn more about this genetic disorder.

Voxzogo is a prescription injectable medicine. Here’s how it treats achondroplasia.

The FDA has approved Crenessity (crinecerfont), the first new CAH treatment in 70 years. Learn how it works and who might benefit.

Duvyzat is a histone deacetylase (HDAC) inhibitor. Here’s how it treats Duchenne muscular dystrophy.

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

Treatment for classic congenital adrenal hyperplasia can have side effects. Here’s what to watch for, including signs of over- or undertreatment like early puberty and weight gain.

What is familial hyperchylomicronemia syndrome (FCS)? Learn about symptoms, diagnosis, treatment, and tips for living with the condition.

Aprende sobre las infecciones por adenovirus. Descubre qué causa estas enfermedades comunes en la infancia y cómo tratar a los niños cuando se enferman.

Achondroplasia dwarfism is the most common form of short-limbed dwarfism. Learn about its causes, symptoms, treatment, and how to care for your child.

Congenital adrenal hyperplasia (CAH) is a genetic condition that affects the body’s adrenal glands. Here’s what to know.

Steroids, salt supplements, and birth control are some of the treatment options for this condition. Learn more about what they do and the potential side effects.

This genetic condition impacts the adrenal glands. Learn more about why it happens, how it affects your child, and more.

Surgery is a good option for pediatric low-grade gliomas. Learn about that and other pLGG treatments, and the side effects your child may have.

What are pediatric low-grade gliomas? Learn what causes these brain tumors and which specialists treat them.

Mirror hand syndrome is an extremely rare birth defect that causes you to have up to 10 fingers without a thumb on one hand. You'll need surgery to fix it.

Caring for a child with Duchenne can be challenging and rewarding. Learn ways to manage the emotional and financial toll it can take on caregivers.