Leadiant Biosciences Ltd. is researching medicines to help people suffering from specific rare diseases, including Cerebrotendinous Xanthomatosis (CTX), Hodgkin Disease (HD), and Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID).

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Metabolic Diseases

CTX

Cerebrotendinous Xanthomatosis (CTX) is a rare genetic metabolic disorder of cholesterol and bile acid metabolism results in systemic and neurological abnormalities. Patients have mutations on the CYP27A1 gene and are characterized by infantile-onset diarrhea, childhood-onset cataract, adolescent- to young adult-onset tendon xanthomas, and adult-onset progressive neurologic dysfunction (dementia, psychiatric disturbances, pyramidal and/or cerebellar signs, dystonia, atypical parkinsonism, peripheral neuropathy, and seizures.

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ADA-SCID

Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID) is an autosomal recessive metabolic disorder that causes immunodeficiency. It occurs in one in 200,000 to 1,000,000 newborns worldwide1. Lack of the enzyme Adenosine Deaminase results in an accumulation of metabolites. The immune system of ADA-SCID patients is severely compromised or completely lacking, and the clinical features are characterized by repeated and persistent infections, which can be severe and life-threatening.

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ADA-SCID

Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID) is an autosomal recessive metabolic disorder that causes immunodeficiency. It occurs in one in 200,000 to 1,000,000 newborns worldwide1. Lack of the enzyme Adenosine Deaminase results in an accumulation of metabolites. The immune system of ADA-SCID patients is severely compromised or completely lacking, and the clinical features are characterized by repeated and persistent infections, which can be severe and life-threatening.

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Cystinosis

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Oncology

Hodgkin Disease

Hodgkin Disease (HD), also known as Hodgkin lymphoma, is a cancer that starts in the lymphatic system. HD originates in white blood cells and breaks the protection against germs and infections. The most common first symptom of HD is swelling and non-painful lymph nodes at neck, armpit and groin, and the spread is through lymph vessels in the whole body with fatigue, weight loss, night sweats and fevers as manifestations.

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Reference:

1 Adenosine deaminase deficiency. Genetics Home Reference; 2016. Available at: https://ghr.nlm.nih.gov/condition/adenosine-deaminase-deficiency. Accessed December 7, 2016.