An 18-year-old Maltese man with LHON disease - a rare eye condition - has been accepted to take part in a clinical trial with nine other patients in a hospital in China’s Hubei province.
This was possible through an agreement reached between the National Alliance for the Support of Rare Diseases and the Chinese authorities, reached with the help of the Chinese Embassy in Malta.
The trial and the agreement were announced on Tuesday by Michelle Muscat, President of the National Alliance for the Support of Rare Diseases, at the launch of this year’s annual campaign for rare disease awareness. The campaign was launched in the Parliament building.
Muscat said this step is offering a glimpse of hope where there was none. The clinical trial includes the administration of a drug the market price of which is about $850,000 (€783,000). The medicine will be given free of charge by the Chinese authorities to the Maltese patient.
Through the same agreement, therapy will be provided to members of the alliance at the Mediterranean Centre for Traditional Chinese Medicine in Corradino. A group of 24 people will start their treatment in the coming weeks.
Muscat said that this year's rare disease awareness campaign will focus mostly on schools to convey information about these conditions and show the value of careers in medicine, science and research. To do this, the first book on rare diseases aimed at primary school students will also be launched.
The NGO hoped it will be invited to take part in discussions on a national strategy on rare diseases.
Rare diseases in Malta
The Malta National Register for Rare Diseases lists about 6,000 patients suffering from a rare disease. However, it is estimated that there are some 27,000 people suffering from a rare disease or condition.
In the EU
- 30 million EU citizens are living with a rare disease.
- There are 6,000 diagnosed rare diseases
- One in ever 2,000 people gets a rare disease.