A global pharmaceutical consultancy and service company has been helping the patients of rare diseases like Duchenne Muscular Dystrophy (DMD) providing them with medicines apart from other therapies.
Known as Ikris Pharma Network (IPN), the company is said to have been assisting at least 58 Indian patients, who have been suffering from DMD, a genetic disorder characterised by progressive muscle degeneration and weakness due to the alterations of a protein called dystrophin that helps keep muscle cells intact.
The company said it has been helping these patients procuring medicines worth Rs 3 crore annually. This is, they said, apart from the coordinated diagnostic support for more than 3500 such patients in the country.
The patients are also getting help from IPN in the documentation of Spinal Muscular Atrophy (SMA) and in procuring gene therapy.
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Some of the existing rare diseases found in the country include haemophilia, thalassemia, sickle-cell anaemia and primary immuno deficiency in children, auto-immune diseases, lysosomal storage disorders such as pompe disease, hirschsprung disease, gaucher’s disease, cystic fibrosis, hemangiomas and certain forms of muscular dystrophies.
Apart from it, the company said, it has helped more than 400 patients who have been suffering from Porphyria, Nephropathic cystinosis, MPS IV, MPS VI, Acromegaly, Achondroplasia, DMS, hyperphenylalaninemia and cushing’s syndrome.
According to IPN, it would also request the Centre for free treatment for those patients who cannot afford expensive treatments for DMD which is more than $ 1.5 million (approximately). Further, the company said it has helped more than 5000 patients worldwide, facing difficulties in their access to rare medicines.
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