Rare Disease Day initiatives from Boehringer, Shire and Genzyme

Hannah Blake

pharmaphorum

Today marks the sixth international Rare Disease Day, led by EURORDIS, to raise awareness for patients with rare diseases in over 60 countries around the world. In coordination with this day, we find out some of the rare disease initiatives launched by Boehringer Ingelheim, Genzyme and Shire.

Boehringer Ingelheim

Boehringer Ingelheim has announced the initiation of a phase 3 clinical trial investigating volasertib, after enrolling its first patient. Volasertib is a selective and potent polo-like kinase (Plk) inhibitor, in combination with chemotherapy, and is being tested to treat patients with acute myeloid leukaemia (AML) who are ineligible for intensive therapy.

Acute leukaemias are rare diseases, with AML being the most deadly acute leukaemia in adults.

Shire

Shire has announced its support for the R.A.R.E. Project, which is a leading non-profit rare disease advocacy organisation, in their Global Genes Project "Wear That You Care™". This is a denim campaign in which denim jeans are used to raise awareness of rare genetic disorders. The company has distributed over 3,500 blue ribbons to employees at various corporate locations worldwide.

"Shire's purpose is to enable people with life-altering conditions to lead better lives - and for us, this includes improving access to information and diagnosis as well as providing much-needed treatment for rare diseases. Rare Disease Day is an occasion to show our commitment to the rare disease community. We hope that this day can highlight the need for consistent, reliable, and effective treatments and services for patients and give hope to those affected by rare diseases."

Sylvie Grégoire, President of HGT.

Genzyme

Genzyme has today launched a new website highlighting collaborative activities taking place globally to recognise Rare Disease Day: http://raredisease.genzyme.com.

The aim of Rare Disease Day is to educate policymakers, medical professionals, patients, caregivers and the general public and to empower them to advocate for those affected by rare diseases. Some of the Genzyme initiatives the company is involved in today include a running and cycling relay race, the launch of “Facing Fabry Together” patient and family rare disease video, a comic book art contest in Hong Kong and an art exhibition in Italy.

Also, Genzyme has launched the third annual Patient Advocacy Leadership Awards (PAL Awards). This is a global grant program that supports non-profit patient organizations that work on behalf of individuals living with lysosomal storage disorders (LSDs), a group of rare, inherited disorders that cause progressive and debilitating health problems.

“Promoting understanding of the journey of those affected by rare diseases while advancing therapies and scientific innovation are core to who Genzyme is, and it is an honor to partner today with advocacy organizations globally to recognize Rare Disease Day. As we begin the PAL Award process again this year, we look forward to seeing how this program can be utilized to support the evolving needs of rare disease advocacy leaders around the world. Together, we recognize that there is still great work – and opportunity – ahead of us.”

Jamie Ring, Genzyme’s Vice President of Global Patient Advocacy for Rare Diseases.