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Hereditary Angioedema (HAE) is an autosomal dominant disease which is caused by a deficiency in functional C1 inhibitor. It is a rare hereditary disease which is characterized by painful, unpredictable, recurrent attacks of inflammation and swelling affecting the hands, feet, face, abdomen, urogenital tract, and the...

Systemic Lupus Erythematosus (SLE) is a chronic, multisystem, inflammatory autoimmune disease which leads to weakening of the immune system. It results in systemic inflammation which affects multiple organs such as kidneys, the tissue lining the lungs (pleura), heart (pericardium), and brain. Acute Cutaneous Lupus, ...

Gaucher Disease is a rare autosomal recessive inherited multisystem disease with phenotypic variation from mild to very severe. It is the most common lysosomal storage disorder which occurs due to the deficiency of an enzyme β-glucocerebrosidase that helps in degrading glucocerebroside into glucose and ceramide. Lac...

Biohaven Completes Enrollment of Rimegepant in Second Pivotal Phase 3 Clinical Trial for the treatment of Migraine Biohaven Pharmaceutical has recently announced the completion of enrollment of their study BHV3000-302 in Phase 3. The company is examining the safety and efficacy of Rimegepant for the treatment of Mi...

Severe Combined Immunodeficiency (SCID) is an inherited primary immunodeficiency disease (PIDD) that typically presents in infancy. It is characterized by profound immune deficiency condition resulting in a weak immune system that is unable to fight off even mild infections. Severe Combined Immunodeficiency is a rar...

Wiskott - Aldrich syndrome (also known as WAS) is a rare X-linked recessive genetic disorder that affects both T-lymphocytes and B-lymphocytes. It occurs due to the mutations caused in the WASp gene and is mainly characterized by low platelet count (microthrombocytopenia), immune deficiency, and eczema. Being an X-...