Building awareness and communities to support and find treatments for Rare Diseases and Genetic Conditions
Once famously described as “orphan diseases, too small to be noticed, too small to be funded” in the Hollywood drama Lorenzo’s Oil, rare diseases are getting unprecedented attention today among drug manufacturers, who are ramping up research efforts and marketing new medicines that promise fuller lives [...]
Shire’s new CEO apprentice has closed on his first buyout deal for the company. Flemming Ornskov took the lead this morning in announcing that Shire ($SHPG) has snapped up the Swedish biotech Premacure AB in a deal that will add a near late-stage, rare disease [...]
There was a time not long ago when patients suffering from rare diseases had little hope of finding a medicine that could cure their ills. But in the 30 years since Congress passed the Orphan Drug Act, research into “orphan drugs” — defined as those [...]
The issue of comparability has long been a topic of strategic discussion in the pharmaceutical industry, and it is now becoming a focus point in the orphan drug space. We have compiled insights on this matter from conversations with industry and from our own [...]
In a recent article (see below), The Burrill Report discusses an increasing interest in orphan drugs, offering promise for treating rare diseases. Life sciences consultant, Kiran Meekings of Thomson Rueters offers her perspective in this essential and impactful podcast: The Burrill Report (August 27, 2012): Feeling Love for [...]
Thanks to the Pew Internet Foundation, we have a lot of data about ePatients. These empowered, engaged and educated patients (and families) are helping to bring about a transformation of healthcare in this country. Pew’s Susannah Fox has named them “healthcare superheroes.” Through our experience [...]
The Month of February During the month of February, much of the American population will be celebrating historical events, special interest groups and campaigns. Some of these include Black History, American History, Children’s Dental Health, and American Heart Month. Internationally recognized events in February include [...]
