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  2. Farber disease - Wikipedia

    en.wikipedia.org/wiki/Farber_disease

    Children who have the classic form of Farber disease develop symptoms within the first few weeks to months of life. [4] Individuals with moderate or attenuated forms may develop symptoms at any time in childhood. Sometimes it is difficult to diagnose Farber disease because the symptoms can be misdiagnosed as Juvenile Idiopathic Arthritis (JIA).

  3. Sphingolipidoses - Wikipedia

    en.wikipedia.org/wiki/Sphingolipidoses

    Enzyme replacement therapy is available to treat mainly Fabry disease and Gaucher disease, and people with these types of sphingolipidoses may live well into adulthood. The other types are generally fatal by age 1 to 5 years for infantile forms, but progression may be mild for juvenile- or adult-onset forms.

  4. Metachromatic leukodystrophy - Wikipedia

    en.wikipedia.org/wiki/Metachromatic_leukodystrophy

    In the late infantile form, which is the most common form of MLD (50–60%), affected children begin having difficulty walking after the first year of life, usually at 15–24 months. Symptoms include muscle wasting and weakness, muscle rigidity , developmental delays, progressive loss of vision leading to blindness, convulsions , impaired ...

  5. List of childhood diseases and disorders - Wikipedia

    en.wikipedia.org/wiki/List_of_childhood_diseases...

    The term childhood disease refers to disease that is contracted or becomes symptomatic before the age of 18 or 21 years old. Many of these diseases can also be contracted by adults. Some childhood diseases include:

  6. Multiple sulfatase deficiency - Wikipedia

    en.wikipedia.org/wiki/Multiple_sulfatase_deficiency

    Multiple sulfatase deficiency (MSD), also known as Austin disease, [1] or mucosulfatidosis, [1] is a very rare autosomal recessive [2] lysosomal storage disease [3] caused by a deficiency in multiple sulfatase enzymes, or in formylglycine-generating enzyme, which activates sulfatases.

  7. GM1 gangliosidoses - Wikipedia

    en.wikipedia.org/wiki/GM1_gangliosidoses

    The GM1 gangliosidoses, usually shortened to GM1, are gangliosidoses caused by mutation in the GLB1 gene resulting in a deficiency of beta-galactosidase.The deficiency causes abnormal storage of acidic lipid materials in cells of the central and peripheral nervous systems, but particularly in the nerve cells, resulting in progressive neurodegeneration.

  8. Lysosomal acid lipase deficiency - Wikipedia

    en.wikipedia.org/wiki/Lysosomal_acid_lipase...

    Older children or adults generally present with a wide range of signs and symptoms that overlap with other disorders. [5] They may have diarrhoea, stomach pain, vomiting, or poor growth, a sign of malabsorption. They may have signs of bile duct problems, like itchiness, jaundice, pale stool, or dark urine. Their feces may be excessively greasy.

  9. Multiple epiphyseal dysplasia - Wikipedia

    en.wikipedia.org/wiki/Multiple_epiphyseal_dysplasia

    Multiple epiphyseal dysplasia (MED), also known as Fairbank's disease, is a rare genetic disorder (dominant form: 1 in 10,000 births) that affects the growing ends of bones. Long bones normally elongate by expansion of cartilage in the growth plate (epiphyseal plate) near their ends.

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