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Gaucher Disease
High unmet needs in the Gaucher disease market calls for a standard and effective treatment option
Gaucher Disease

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Retinitis pigmentosa Newsletter
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Retinitis pigmentosa is a gradual deterioration of the ability of eyes to see. The defect in the eye is inflicted upon the light-sensitive tissue...

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Chronic Pancreatitis is an inflammation of the pancreas that can be a result of calcium stones and cysts, acute inflammation, cystic fibrosis, unhealthy lifestyle habits, certain autoimmune disorders or due to hereditary diseases...

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Chronic inflammatory demyelinating polyneuropathy market

A rare neurological disorder characterized by progressive weakness and impaired sensory function in the legs and arms...

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Paroxysmal Nocturnal Hemoglobinuria

Paroxysmal Nocturnal Hemoglobinuria (PNH) is a rare acquired disease that leads to the destruction of red blood cells...

Pulmonary Arterial Hypertension (PAH)
Pulmonary Arterial Hypertension (PAH)

Pulmonary Arterial Hypertension (PAH) is a rare, chronic, and progressive form of Pulmonary Hypertension, delineated by the elevated pulmonary arterial pressure (PAP) and pulmonary vascular resistance (PVR) in...