Rare Disease Medicines:
Global Access to Specialized Treatments

Rare disease medicines (also called orphan drugs) are often approved in the US, Europe or Japan years before they reach India, or are never launched here at all. Named Patient Program helps doctors, hospitals and patients get these specialised rare disease treatments through the legal named patient import route. Below are the rare disease medicines we can help source, including therapies for Duchenne muscular dystrophy, phenylketonuria (PKU), Batten disease (CLN2), MPS IVA and VI, achondroplasia, Gaucher disease and Cushing's disease.

Amondys 45

Brineura

Carbaglu

Cystagon

Epidiolex

Exondys 51

Isturisa

Kuvan

Luvometinib

Naglazyme

Normosang

Tepezza

Translarna

Velaglucerase Beta

Vimizim

Voxzogo

Vyondys 53

Zavesca

Advances in medical science are helping to develop new therapies for rare diseases, but these treatments are not always available in every country. In many cases, patients and healthcare providers must look beyond local markets to access specialized medicines. If a treatment approved by a recognized medical authority is not available in your country, it may be possible to access it through our service. With a valid prescription from your doctor, we may be able to source and deliver the medicine through compliant patient access programs.

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Rare Disease

Rare Disease Medicines: Global Access to Specialized Treatments

Frequently AskedQuestions

Rare Disease Medicines:
Global Access to Specialized Treatments

Frequently Asked
Questions