The IPA has authored an article titled “The role of patient advocacy organizations in shaping medical research: the Pompe model,” and we are excited to announce that it has been accepted for publication.

The article focuses on the Pompe Model, a term describing the long-standing relationship between the patient community, the medical/scientific community, and industry. Over the last 20 years, we have seen that as a united community of patients, family, friends, physicians, scientists, and industry, working together, we have an undeniable strength and we have made, and will continue to make, great strides in learning more about Pompe Disease and in working towards treatments and disease management.

The article is published in the Annals of Translational Medicine, an international, peer-reviewed  journal, and can be found here.

Ride With Hope is a campaign organized by the MLDA, Malaysia Lysosomal Diseases Association, a member of the International Pompe Association, on the initiative of the MLDA president, Ir Lee Yee Seng.

The objective of the campaign is to raise funds, in addition to raise international awareness on Lysosomal Storage Diseases and other rare diseases, including Pompe disease.

Three Malaysian riders, Ir Lee Yee Seng, Faye Tan and Ben Chong Jun Keong will be taking the lead in this challenge, they are challenging themselves in the pursuit of helping rare disease children in Malaysia by initiating the Ride with Hope initiative. The MLDA will be working together with Audax Club Parisien and with Paris-Brest-Paris (PBP), a 1200-kilometer bicycling event held every 4 years, a rare opportunity for patient organizations to work together in a collaborative manner.

There will be 6,600 riders from all around the world participating in this event and patient organizations can look for their own country riders by searching in the FB close group “AUDAX Randonneurs Country XXX” or visiting Audax Club Parisien page http://www.audax-club-parisien.com/EN/# for further enquiry on their country riders.

Each rider will ride together with Hope, which is the mascot of this event. The riders need to complete the entire journey together with Hope, despite the 11,000 m of elevation, which will test the endurance level of participants, just like one of the many challenges of living with a rare disease. MLDA initiated Ride with Hope and is willing to share all the resources with all the rare disease patient groups from all around the world including supplying Hope, logo and other products.

If every participant can help the MLDA raise RM 1,000, then 1,000 people can successfully raise up to RM1 million throughout this campaign. Raised funds will be distributed for patient welfare and subsidies, medicine purchase, genetic screening, medical equipment, and for MLDA future activities.

Participants can join the Ride with Hope campaign sharing their fund raising activities on the Ride with Hope FB page; they can walk, run, ride, hike, do charity sales and many other activities, and make it as viral as possible. Through this, we are also able to educate the younger generation that not everything in life is easy. There are many things we have to feel blessed for, and the main one is health. With more awareness on rare diseases, teachers and counsellors will be more sensitive towards the needs of rare disease patients while attending their schooling.

Awareness is the key to unlock the assistance and help this special group needs in order to survive and live a life as normal as possible. Without awareness people will question the allocation of budget by the Malaysian Ministry of Health, peers will laugh at rare disease children in school for having a disability or using a wheelchair, parents will face hardship in work as they need to bring their children to ERT at a weekly or fortnightly basis, parents will also blame themselves for something which they cannot control and many more struggles we cannot foresee.

The success is when the country’s population is aware of rare diseases and will not sideline the patients and families. We have a long way more to go to achieve the level of awareness that we are hoping for.  We as a non-profit organization must continue to request corporations to collaborate with us to organize big events such as this to raise awareness and also generate a sustainable funding for expensive treatments.    

With all the patient organization participating together, this will help to create greater awareness and spread out through the social media and this will not only help raise, but also reach the attention of the policy makers. Ride with Hope carries the clear message that in every country in this world, we should leave no one behind.

Ir Lee Yee Seng is the  President of the Malaysia Lysosomal Disease Association (MLDA), he is also the father of three children, two of whom have Pompe Disease. The MLDA was established in 2011 to advocate for patients’ rights to a sustainable health care and support system, and raise the awareness of the general public about life-threatening Lysosomal Storage Diseases. This small band of advocates have made great headway in bringing rare diseases to the attention of policymakers, the media and health care providers. Lee’s exuberance is contagious as he shares his life’s experiences, joys and sorrows. In 2014, he was awarded the My Hero Award in Malaysia. The treatment for Pompe disease is expensive and it is not readily affordable by everyone. However, Lee feels strongly that the dignity of life should be respected. The challenge may be an uphill task, but its cause is championed by people who have the heart of compassion to move mountains for even one child to receive the gift of a new life. The human will is an extraordinary mechanism and Lee’s own experience with his daughters overcoming Pompe is really a message of hope to all.

April 15, 2019

Today is our 6^th International Pompe Day!

The goal of International Pompe Day is to foster international awareness of Pompe Disease and the slogan for our day is Together We Are Strong.

The IPA believes that raising awareness about Pompe Disease is key, and for 2019 the theme that has been selected is Moving On with Pompe: an encouraging and positive theme to show how it is possible to continue living a fulfilling life after diagnosis and despite the challenges of living with Pompe disease, in the hope of inspiring others.

Inspiring stories from our international community have been collected in a dedicated blog that the IPA has specifically created: https://ipompeday.blogspot.com

Please read and share these stories, so we can inspire others and show the world how strong our Community is!

Thanks to all that have contributed with their story, and happy International Pompe Day!