Rare diseases
For a person suffering from a rare condition like Hemophilia, the experience is very real.
For a person suffering from a rare condition like Hemophilia, the experience is very real.
But for many people who are not intimately familiar with rare or ultra-rare diseases and conditions, it can be a matter of “out of sight, out of mind.”
Even the definition of “rare” differs from country to country; for example, in the US a rare disorder is one that affects fewer than 200,000 Americans, currently about 1 in 1,700 people*, while the European Commission on Public Health defines rare disease as a “life-threatening or chronically debilitating diseases that affects 1 in 2,000 people**”. Neglected by public attention, lacking neither a path to a clear return on investment for pharmaceutical companies nor a constituency large enough to readily draw political notice and public research support, rare diseases are sometimes called “orphan diseases”.
* Rare Diseases Act of 2002, [Page 116 Stat. 1988], Public Law 107-280, 107th Congress
** Decision No 1295/1999/EC of the European Parliament and of the Council of 29 April 1999 adopting a program of Community action on rare diseases within the framework for action in the field of public health (1999 to 2003)
A Real and Continuing Journey of Science and Humanity
When we say “Rare is Real”, we mean to remind everyone not suffering from these rare conditions that they deserve attention.
And we remind ourselves that we, at Kedrion, have an opportunity to serve an often-neglected population of patients.
For Kedrion, rare diseases and disorders have always been real, and a focus.
Hemophilia, Primary Immunodeficiencies, Plasminogen Deficiency, and Chronic Inflammatory Demyelinating Polyradiculoneuropathy are among the conditions our plasma-derived therapies treat and we have long worked with governments, caregivers and advocacy groups to encourage awareness to these health challenges.
These efforts are confirmed by the never-ending commitment to providing relief to patients with an expanding portfolio and expertise. This is particularly true when we stabilized supply of RYPLAZIM® in the US, marking an important moment for patients affected by an ultra-rare condition.
Rebecca Bialas
MD, Co-Founder and Chair of the Plasminogen Deficiency Foundation
While virtually all genetic disorders are rare, not all rare diseases are genetic.
An example of the latter is Rabies*, an extremely deadly, but fortunately rare disease Kedrion is committed to helping prevent. Another is Hemolytic Disease of the Fetus and Newborn (HDFN).
* Source: https://rarediseases.org/rare-diseases/rabies
Hear the voice of one of our patients, Christine.
Hemolytic Disease of the Fetus and Newborn is a rare condition where an Rh-Negative mother carrying an Rh-Positive fetus may develop antibodies that harm the fetus*.
A prophylaxis* introduced in 1968 can prevent maternal immune sensitization using plasma donors sensitized like Christine. Christine, a nurse practitioner and sensitized Rh-Negative donor, exemplifies what we call the "Circle of Care", from the experience of the disease to the decision to help protecting other pregnant women and their fetuses from HDFN. She recounted her inspiring story during a “Patient Day” at the Kedrion facility in Melville, NY, where the HDFN prophylaxis is produced.
* Source: https://www.ncbi.nlm.nih.gov/books/NBK560488
