Genzyme Rare Community

Has it been a while since you thought about the basics of biology? The Massachusetts Institute of Technology (MIT) and Harvard University teamed up with Eric Lander, one of the leaders of the human genome project and founder of the Broad Institute, to provide a free online course in biology. It promises to be a fun and freewheeling introduction to “the greatest scientific revolution of the last century.”

The 12 week class, “Introduction to Biology – The Secret of Life,” will be available free online via edX starting March 5th. Details can be found on the edX website.

In addition, National Public Radio talked to Mr. Lander about the course and you can click here to listen to the interview.
 

PAL Award recipient FSIGN (the Dutch Fabry Support Group) recently declared the first Saturday in April as the annual International Fabry Women’s Day. The goals of the day are to increase recognition and support for all women with Fabry Disease and those directly affected by Fabry Disease such as partners, family members and friends.

For the past seven years, FSIGN has organized a national Fabry Women's day for their female membership, enabling them to come together and share experiences, exchange information and provide support to one another. Due to its success, FSIGN is now working to make the day global. “Our hope is that on this day, activities will be organised for Fabry women. It doesn’t matter if these are large of small as everything helps,” says Erica Schenk of FSIGN.
 

We were delighted to present two 2012 Patient Advocacy Leadership Award recipients in Asia with their awards in Beijing, China this past fall. The Hong Kong Mucopolysaccaridoses and Mutual Aid Society (HKMPS) and the Genetic LSD Foundation of Thailand were on hand at the Rare Disease Multi-Stakeholder Forum to participate in an award ceremony recognizing their achievements through the PAL Awards. As part of their PAL Award, both groups have received grants of $10,000 each. Also recognized was Kenneth Mah of Singapore, whose organization Rare Disease Disorders Society (Singapore), received a 2011 PAL Award.

Mr. Stephen Ma of the HKMPS and Mr. Boon Putthipongtanachot of the Genetic LSD Foundation received the awards on behalf of their respective organizations. The HKMPS PAL project will involve the creation of an art competition among high school and college students that would illustrate, in comic format, what it is like to live with MPS based on real-life stories. The comics will then be compiled into a small book which will be made available for a donation to the society. In Thailand, the Genetic LSD Foundation PAL Award will support the creation of a regional patient group network to reach underserved patient populations in remote regions of the country.

Genzyme congratulates both organizations on their PAL Awards!

Pictured from left to right: Yoshikazu Nakamura, Regional President of Japan-Asia Pacific, Genzyme; Boon Putthipongtanachot of the Genetic LSD Foundation; and Rogerio Vivaldi, Senior Vice President and Head, Rare Diseases Genzyme; Carol Tan, Vice President and General Manager of Sub-Asia Region, Genzyme; Stephen Ma, Hong Kong Mucopolysaccaridoses and Mutual Aid Society; and Cara Hesse, Genzyme.

Attendees at the National Society of Genetic Counselors (NSGC) annual conference learned about a unique way for high school students to understand the science behind rare diseases. Julia Su, a genetic counselor from Canada, created a biology curriculum with the help of the National Organization for Rare Disorders (NORD). Utilizing a “train the trainer” model, Su devised lesson plans that captured the experiences of approximately 700 affected individuals, families, health care professionals, patient organization advocates and others. Topics covered in this curriculum include an introduction to rare diseases, scientific background, individual stories and the related ethical, legal, and social issues.

Available for downloading from http://rarediseaseday.us/, more than 600 students have already benefitted from this curriculum. You can learn more about Julia's project by reading her blog post here. A perfect companion piece to the NIH ORDR’s Middle School rare disease curriculum, patient advocates could utilize this reliable educational resource for local awareness as well as incorporating it into a Rare Disease Day event in the future.

For more information about Genzyme's support for International Rare Disease Day, you can read our press release here.

Each year, for the past 6 years, our team has been involved in the planning and execution of Genzyme’s recognition of Rare Disease Day. Beginning with a small breakfast in 2008 for colleagues Massachusetts, we’re delighted that this year’s signature event – a relay race with an Olympic style torch -- will now include colleagues in the Netherlands and Ireland. Every year, Rare Disease Day gets bigger and better!    

Since rare diseases are at the heart of Genzyme, Rare Disease Day has a very special place for all us in our corporate headquarters in Cambridge, Massachusetts. Last year, our celebration grew to become a full-days’ worth of activities that revolved around a relay race between several Massachusetts campuses of Genzyme. The relay idea came out of our desire to ensure that the sense of connection and inspiration that comes from Rare Disease Day reached as many Genzyme employees as possible.

This year’s event will start at the Genzyme office in the Netherlands, with an 18 kilometer tandem cycling event that will travel from Naarden to the offices of the Dutch neuromuscular patient organization, VSN. Once cyclists in Naarden complete their leg of the relay, they will pass the torch – via live video link – to colleagues in Waterford, Ireland who will run another leg of the relay. Once their leg is complete, a second passing of the torch will occur between Ireland and Massachusetts. All of these events will include representatives from the patient advocacy community as well as individuals affected by rare diseases.

Rare Disease Day continues to motivate, inspire and connect us at Genzyme not only as colleagues joined by our profession, but as individuals with a desire to participate in a vast global community dedicated to improving the lives of people affected by rare diseases. In our view, this year’s theme, “Rare Disorders without Borders”, couldn’t be more appropriate. Please click here for more information about Genzyme’s involvement in Rare Disease Day.

Last night, Jamie Ring and I had the great opportunity to attend the EURORDIS Black Pearl Gala Dinner in Brussels, Belgium. During the evening’s ceremony, Genzyme was presented with the EURORDIS Company Award which recognizes the outstanding accomplishments of companies dedicated to rare diseases. Genzyme was honored for pioneering the development and delivery of therapies for rare diseases, its longstanding support of patient organizations, including EURORDIS, as well as initiatives to increase patient access to Genzyme treatments.  Genzyme’s Senior Vice President of Rare Diseases in Europe, Hilde Furberg, accepted the award on behalf of Genzyme. It was made all the more special that we were joined by some friends from the rare disease advocacy community at our table. Thank you to the EURORDIS team for this wonderful acknowledgement. There is more work to be done of course, but we greatly appreciate the recognition. For more information, please see Genzyme's press release.

Like many of you, we’ve been busy planning for Rare Disease Day next week and we’re looking forward to kicking off our activities. We’ve heard from our partners about some of the amazing activities being planned to celebrate, empower and mobilize this vast global community. Hearing about your plans reminds us that regardless of where we live in the world -- or which disease we are focused on -- Rare Disease Day is our one day to all come together in a truly visible and powerful way.

To support this year’s theme, “Rare Disorders Without Borders”, Genzyme launched a new website  http://raredisease.genzyme.com/ that pulls together the many activities we will be involved in around the world, together with our partners in the advocacy world and in government. If you aren’t yet involved in a Rare Disease Day activity, visit www.rarediseaseday.org and find one to join! 

Like many of you, we are well into our planning for Rare Disease Day 2013 activities! This year’s theme, Rare Disorders Without Borders, is a powerful message that I know all of us in the rare disease space can appreciate. As an organization with offices in more than 50 countries supporting patients in over 100 countries, we can attest to the global nature of rare diseases and we know how important it is to raise disease awareness EVERYWHERE! 

Watching the evolution of Rare Disease Day from its inception has been truly amazing. Even at Genzyme, our activities have evolved from a small Rare Disease Day breakfast in our Cambridge office the first year to now hosting activities in most of our local offices around the world. This year, our advocacy team will be spreading out on Rare Disease Day participating in events in Belgium, Ireland, the Netherlands, Massachusetts and Nevada. Stay tuned for more updates about our activities!  

This post comes to us from Adi Melcer from the Genzyme-Israel office. Adi, along with several of his Genzyme-Israel colleagues, attended the Israeli Gaucher Patients’ Association annual meeting on January 1st. Here Adi describes the meeting and the response in Israel to the green shoe lace campaign.

My colleagues and I were delighted to participate in the annual meeting of the Israeli Gaucher Patients’ Association held in early January. More than 250 people attended the conference, including patients, families, clinicians, researchers, industry representatives and many others. It was a packed agenda, with workshops on topics such as patients’ rights and updates on recent changes to the Ministry of Health committee that reviews treatment for Gaucher disease. 

This year we decided to follow the lead of our U.S. counterparts by initiating the “Steps Ahead of Gaucher” campaign in Israel. Genzyme launched the campaign last year to support the efforts of the U.S.-based National Gaucher Foundation. We thought this was a great initiative that would also resonate with the Israeli Gaucher patient community, so we launched it at the meeting and invited attendees to join us in “lacing up.” Though our goal was to help increase awareness in Israel and support the Israeli Gaucher Patients’ Association, of course, we were delighted to also help make this campaign a more global one by bringing it to Israel.