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Jan-17-2010 20:20printcomments

Extraordinary Measures the Movie: A Discussion by Pompe Disease Support Groups

As a community we hope that this film will lead to a greater awareness of Pompe disease and thereby improved diagnosis of the condition.

Scene from Extraordinary Measures the Movie
Scene from Extraordinary Measures the Movie
Courtesy: extraordinarymeasuresthemovie.com

(SAN ANTONIO, Texas) - Extraordinary Measures is a new movie scheduled for release in America on January 22, 2010. Starring Harrison Ford and Brendan Fraser it tells the story of a family struggling with a rare disease-­‐ the neuromuscular condition known as Pompe Disease (aka Acid Maltase Deficiency).

The Acid Maltase Deficiency Association (AMDA) is a US patient support group for sufferers of Pompe Disease. The International Pompe Association (IPA) is a federation of nearly 40 national patient groups around the world who collectively support over 1000 patients and their families.

Pompe disease is an extremely rare, progressive neurological condition that affects people of all ages; the classical infantile form is especially severe as it particularly damages the heart, respiratory muscles and skeletal muscle.

Older infants, children and adults are affected to varying degrees and without treatment all may suffer from loss of muscle function and breathing difficulties. Quality of life is reduced and life expectancy is considerably shortened; most untreated infants do not live beyond their first birthday.

Every patient has their own unique story, and every family is affected in its own way.

Extraordinary Measures

The film Extraordinary Measures focuses on the actions of one highly-­‐motivated individual and his collaboration with a medical researcher. It shows how Pompe disease, a devastating illness, changes family life forever, something that is true whatever the severity or age of the sufferer.

It is a remarkable story about the man and his quest to find a treatment for his children – but it is NOT the story of the development of a treatment for Pompe disease; that is a misconception implied by the film and spread by other commentators.

Trailer for Extraordinary Measures

The real story behind the development of an effective treatment involved close collaboration between families, researchers and industry and was underway long before John Crowley’s involvement.

The presence of his company did not lead to the development or acceleration of a therapy for Pompe disease; that process was already making good progress elsewhere.

The missing part of the Hollywood story is that the development of a treatment involved an international Pompe community; this has been airbrushed out of the picture by the movie and the associated book. Since the early 1990’s the AMDA and other IPA affiliated organizations have together been very closely involved with the research and subsequent development of an Enzyme Replacement Therapy (ERT) for Pompe disease.

We have long worked with the Dutch team of researchers at the Erasmus Medical Centre (Rotterdam). These trail‐blazing pioneers proved the principle of ERT for Pompe disease, carried out the first successful clinical trial and led the way towards a commercial therapy. They did so on a shoestring budget, in the face of indifference from the medical establishment. It is therefore particularly disappointing that no mention is made of their contribution.

It is apparent by the end of the movie that the therapy is certainly not a miracle cure and it is for that reason that the Pompe community, through the AMDA and IPA, continues to work with researchers and industry towards future therapies. However, it is important to remember that ERT is helping hundreds of patients around the world today.

Research and patient communities fighting rare diseases such as Pompe yearn for publicity to help them reach out to the public, patients, families, medical professionals and politicians, and for that reason we are very grateful to this movie.

As a community we hope that this film will lead to a greater awareness of Pompe disease, and therefore improved diagnosis of the condition. We hope that the information about the disease will help families quickly find support from both medical professionals and patient support groups, and that screening programs to accurately identify the disease will be given a high priority.

We hope the community at large will realize the critical importance of newborn screening – by far the best way to identify the disease so that timely treatment can limit irreparable muscle damage, giving those affected the best opportunity to lead a normal life

Recommended Reading

IPA founding member Kevin O’Donnell is currently writing a chronicle of events that led to the development of the commercial Enzyme Replacement Therapy for Pompe disease. “Pompe Disease – The Real Story” can be read online at: pompestory.blogspot.com

Whilst we would recommend that you read the whole story, if time is limited there is a search facility to locate subjects of interest.

Movie link: extraordinarymeasuresthemovie.com/a>


The AMDA and IPA's representatives can be accessed below:

AMDA Tiffany House, President AMDA (Acid Maltase Deficiency Association) P.O. Box 700248 San Antonio, Texas 78270-­‐0248 USA Email: TiffanyLHouse@aol.com Website: amda-­pompe.org

IPA Allan Muir, Chairman IPA (International Pompe Association) Lieutenant Generaal van Heutszlaan 6 3743 JN Baarn The Netherlands Email: allan@pompe.org.uk Website: WorldPompe.org

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Ed Madara January 18, 2010 12:27 pm (Pacific time)

The point is well made how it's been self-help support groups of families and individuals with the disorder have been the first ones to actually advocate and fight for needed research, treatment, both public & professional education, as well as often even the first recognition of the disorder. That's been, and unfortunately remains, the real story. For information on most any rare disorder: National Organization for Rare Disorders http://www.rarediseases.org A genetic disorder? In the USA, there's The Genetic Alliance (formerly the National Organization of Genetic Support Groups http://www.geneticalliance.org To start a needed new rare disorder mutual support network that doesn't yet exist, there's the American Self-Help Group Clearinghouse: www.selfhelpgroups.org

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Sean Flynn was a photojournalist in Vietnam, taken captive in 1970 in Cambodia and never seen again.