2009-07-28
Two patients with MPS VI began ERT

Others still pending government approval

After years of efforts and hard work, we received good news from HA on May 15, 2009, which was “International MPS Day” and the day after our hosting of the 2009 Asia-Pacific MPS Conference, that the 2 children with MPS VI were approved to use ERT. HA will enter them into a one-year research trial, with the enzyme sponsored by drug company, in hopes of stabilizing their cardiorespiratory functions and preventing their condition from getting worse.

The 2 children have begun receiving their ERT treatment in July, visiting the hospital for intravenous injection of 4 to 5 hours every week.

This is a small step forward by Hong Kong for MPS patients! We still have some journey to go to achieve “one-stop shop” integrated treatment (including diagnosis, assessment, treatment and rehabilitation) for all patients.

As for other patients, for example, 2 out of the 3 patients with MPS type I are suitable for ERT. After our discussions with the drug company that provides the enzyme for MPS I, the 2 patients were promised ERT sponsorship for half a year each. In addition to the help of many kind-hearted donors over these 2 years, we have secured sufficient funds for both of them to receive a full year of ERT.

The enzyme to treat MPS I was the first ERT treatment available to patients, with evident efficacy overseas. The Food and Health Bureau Chief Dr York Chow has acknowledged that MPS is one of over fifty rare genetic lysosomal diseases with actual medical needs. Since the year 2008-09, the Hospital Authority (HA) has also received additional annual recurrent funding of $10 million to provide ERT to patients with rare genetic lysosomal diseases.

Since the 2 children with MPS VI can receive ERT, it only makes sense for the 2 children with MPS I, who are younger and with less severe illness, to receive treatment as soon as possible. ERT can help prevent their health from getting worse or leading to other problems, such as weaker heart and lung functions and rigid joints. Yet, as long as HA does not approve them for using ERT, they cannot begin treatment.

We hope HA will ratify all patients’ therapeutic needs as soon as possible

2009-05-15
International MPS Day

2009-05-12 to 2009-05-14 
2009 Asia-Pacific MPS Conference

Our Right To Treatment

We hosted a conference in May, with invitations to sister organizations in the Asia Pacific region to share experience on their process about advocating for medical treatment for their rare genetic diseases. We also invited local patient groups to attend.

Details
Joint Declaration
Photo Gallery

2009-03-08 (Sunday)
Buddies Scheme Kick-off Ceremony

The Buddies Scheme is the first program ever in Hong Kong where you can pair-up one-on-one with patients with rare genetic diseases. The scheme aims to connect MPS patients and the public at large as buddies, gain financial support from the community to cover over the million-dollar medication cost per patient each year, and raise awareness to rare genetic diseases as well as the needs of patients who live with such diseases. It is co-organized by our group and final year students majoring in English for Professional Communication at the City University of Hong Kong.

You are cordially invited to attend the official Buddies Scheme kick-off ceremony:

For details about the “Buddies Scheme”, please click here.