But data for rare diseases can be difficult to obtain, which is why patient registries are playing an increasingly important role in helping bridge the data gap.
By Ashley Winslow, Ph.D.
October 25, 2017
The University of Pennsylvania Orphan Disease Center’s (ODC) international patient registry is a collection of information about individuals focused around a specific diagnosis or condition. The registry’s goal is to generate better data, which can result in better trial design, quicker recruitment of patients and faster approvals of new drugs.
We appreciate and thank all our loyal CSL Plasma donors. Without their plasma donations, we would not be able to make our lifesaving therapies for the treatment of rare and serious diseases.
October 4, 2017
Hereditary angioedema (HAE) is a rare genetic disease caused by a deficiency or dysfunction of a protein called C1 esterase inhibitor. HAE can cause swelling in certain parts of the body, such as the stomach, hands, and face, which can be painful and, in some cases, life threatening.
August 31st, 2017
Margaret Mary Conger, a member of CSL Behring’s Patient Engagement and Reimbursement team, recently sat down with Tad Rockwell, hereditary angioedema patient, clinical trial participant and passionate advocate, who spoke candidly about his long and scary road to diagnosis and treatment.
Tad Rockwell, who battled hereditary angioedema since he was a child, enjoys making breakfast for his family.
By Dr. Lutz Bonacker
August 24, 2017
At CSL Behring we have been working collaboratively with patient organizations for years, informed by our core value that patients come first. Our CEO Paul Perreault puts it this way: “If you take care of the patients, the business takes care of itself.” It has also been our experience that the sole commitment of every patient group with which we collaborate is to the health and well-being of their members.
Researchers are targeting autoimmune epilepsy and chronic inflammatory demyelinating polyneuropathy.
By Karen MacPhail and Gabriela Espinoza, MD
July 25, 2017
As many as one billion people worldwide suffer from more than 600 neurological disorders, according to the World Health Organization and University of California, San Francisco, respectively. Of that number, it is estimated that 6.8 million people die each year from neurological disorders ranging from epilepsy to Alzheimer’s disease.
Neurological disorders affect the central nervous systems or the peripheral nervous systems and can impair the brain, spinal cord, peripheral nerve or neuromuscular function.