Fleming Europe giving hope to DMD patients
"FEW months following Misko's birth in January 2007, a blood test revealed that his liver enzymes were elevated but his parents did not consider it extremely serious. Eventually the baby boy was diagnosed with Duchenne Muscular Dystrophy (DMD), an inherited disorder that causes rapidly progressive muscle weakness and wasting. The child will be wheelchair-bound by the age of 10, and he will most likely not live past his 20th birthday," says story by EURORDIS, first published in July 2011.
Though few patients suffer from this rare disease, Misko is far from the only one. According to DMDfund.org, approximately 1 in every 2,400 boys worldwide is born with Duchenne Muscular Dystrophy. In contrast to cancer, it is uniformly fatal.
Could conducting of more clinical trials and intensified research be the way leading to decrease of the death rate of this incurable disease? What are the ways leading to increased R&D of orphan drugs?
To answer these questions and give DMD patients a hope, Fleming Europe is organizing its 3rd Annual Orphan Drugs Congress on 21 - 22 May 2014 in Barcelona.
Over two days, worldwide Pharma specialists will focus on building up efficiency in rare diseases clinical development programs, patient registries and databases as a key instrument for the development of clinical research, enhancing the all-important collaborative efforts at the regional, national and international levels, and much more.
Attendees will gain a better understanding of the interpretation of orphan drugs legislation and its impact on drug development, learn how to improve processes for designation and speed up approval processes, and examine the essential role of patient organisations in promoting access to treatment.
For the detailed event program and more information about Fleming Europe's 3rd Annual Orphan Drugs Congress, download the event brochure.