Health TopicsGenetic disorders and birth defects

Genetic disorders and birth defects

Find out everything you need to know about genetic disorders, including symptoms, treatment, causes, management and more.

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Treatment for paroxysmal nocturnal hemoglobinuria (PNH)
How medications called monoclonal antibodies, anticoagulants, and stem cell transplants are used to ...

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ISM: tips for building your healthcare team
The healthcare providers that treat indolent systemic mastocytosis (ISM), with tips on finding a spe...

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What are the treatment options for sickle cell disease?
An overview of the therapies used to treat sickle cell disease, including bone marrow transplants an...

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How can Duchenne muscular dystrophy be treated?
Get an overview of the treatments for this common form of muscular dystrophy, including exon-skippin...

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Taking care of mental health when living with PNH
Strategies for coping with the stress of living with a chronic condition like paroxysmal nocturnal h...

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Living with primary immunodeficiency: building your support system
Strategies for coping with the challenges of living with these rare disorders that cause immunodefic...

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CAH and taking care of yourself as a parent or caregiver
Strategies for managing stress and avoiding caregiver burnout when caring for a child with congenita...

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What is the treatment for spinal muscular atrophy?
Learn about the medications and therapies that can help slow the progression of spinal muscular atro...

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5 resources for people with spinal muscular atrophy
Use these links to learn more about spinal muscular atrophy (SMA), including patient education and s...

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Coping with the emotional impact of SMA
How to find healthy ways to cope with the mental and emotional challenges of living with spinal musc...