The biotechnology company has tested its two drugs namely budesonide and maralixibat against two rare diseases in the phase-II trial.
US-Based Company to Acquire a Rare Disease Company 'Scioderm' in Durham For $229 Million
Amicus Therapeutics a champion in the field of rare disease along with Scioderm aims to bring in a treatment for patients with epidermolysis bullosa in the US.
Amicus Therapeutics a champion in the field of rare disease along with Scioderm aims to bring in a treatment for patients with epidermolysis bullosa in the US.
‘Shire has been granted a breakthrough designation from the FDA for its two experimental drugs budesonide and maralixibat for rare diseases eosinophilic esophagitis and progressive familial intrahepatic cholestasis type 2 respectively.’
Eosinophilic Esophagitis is a rare immune system disease occurring due to the accumulation of eosinophils, a type of white blood cells, in the lining of the esophagus, resulting in inflammation. Budesonide has shown positive results against the disease in the phase -II trial. Now, the company is testing the drug in adolescents and adults ages 11-55 years with EoE in the phase-III trial.
Progressive familial intrahepatic cholestasis type 2 is a group of autosomal-recessive liver disorders developing during infancy. It usually progresses into cirrhosis The experimental drug maralixibat has reduced serum bile acids, skin itching, leading to normalization of liver in some patients tested in the phase-II trial.
Popular Social Media Sites Can Act as Platforms for Sharing Information on Rare Diseases
Digital media eases the process of health knowledge sharing and creates new ways to access health information on rare diseases.
Digital media eases the process of health knowledge sharing and creates new ways to access health information on rare diseases.
Funds and Timely Treatment Crucial for Tackling Rare Diseases in India
Lysosomal Storage Disorders occur in about one in 5,000 live births. India currently has about 300-400 patients who have been diagnosed with treatable LSDs.
Lysosomal Storage Disorders occur in about one in 5,000 live births. India currently has about 300-400 patients who have been diagnosed with treatable LSDs.
Coordinated Effort is Important in Fight Against Rare Diseases
Since there are very few cases of rare diseases reported in India, health ministries do not make an effort to know about them, says APJ Abdul Kalam, former President of India.
Since there are very few cases of rare diseases reported in India, health ministries do not make an effort to know about them, says APJ Abdul Kalam, former President of India.
