Charitable Access Programs

Charitable Access Programs – A Lifeline !!

Many MPS and related diseases patients and their families, my family included, benefit from the advances in science and receive the ground-breaking treatments and therapies. Not only are we able to have access to these treatments and therapies, but we also are fortunate to have world leading standards of clinical care thanks to the healthcare systems in many of our countries.

Imagine a world where you or a loved one has been diagnosed with an MPS or related condition, knowing there is a treatment or therapy in other parts of the world, but because you live in a certain country, this treatment or therapy is not available. For many families this is a stark reality, a world without hope of treatment and/or little healthcare.

But there is some hope through the Charitable Access Programs. The possibility of gaining access to certain therapies or treatments despite their unavailability through the healthcare system in that particular country.

Two access schemes sponsored by Takeda and Sanofi Genzyme offer a glimmer of hope to a few individuals and their families who meet the treatment criteria. These Charitable Access Programs offer Enzyme Replacement Therapy options for some Lysosomal Storage Diseases including Gaucher, Fabry, Pompe and MPSII. Currently some of the countries (by no means all) that these treatments are available in on a limited charitable basis are listed below:


The International MPS Network (IMPSN) is actively involved in the Charitable Access Programs and we are diligently remaining current on other countries where these treatments and therapies will be accessible through the program. The Access Program is about available treatments, having access to basic healthcare, and support through patient organizations. The goal is to provide a safe and accessible system. The IMPSN will continue to provide current information and be actively involved in the Charitable Access Programs. Our hope is that more individuals will be commissioned over the coming months and years by additional industries collaborating and finding mechanisms for involvement.

Although these programs are not available for everyone, they demonstrate that it is possible to innovate and give hope. This is a path of true advocacy.  A founding principle of all our patient organisations and one we are rightly proud of.

For further information please follow the link below or contact Bob Stevens, Group CEO UK MPS Society at

For more information visit:


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