"FWGBD is very pleased to report that our founding President, Dr. Andra H. James (Andi), has been awarded the HTRS (Hemostasis and Thrombosis Research Society) Lifetime Achievement Award, at their 2017 Scientific Symposium on April 8, 2017.
Andi is an Ob/Gyn with a singular grasp of benign hematology. Her understanding of, and concern about bleeding and clotting disorders in women was informed and enhanced by her Fellowship in Maternal-Fetal Medicine at Duke, where she spent three years in the laboratory of Dr. Russell Ware; her Ob/Gyn residency at the University of North Carolina-Chapel Hill, incubator of advances in knowledge about hemophilia and hemostasis; by her graduate training in Public Health at Johns Hopkins; and by having been a nurse-midwife at the start of her career."
- From the Foundation for Women & Girls with Blood Disorders
Breda, the Netherlands / Ghent, Belgium
argenx, a clinical-stage biotechnology company developing a deep pipeline of differentiated antibody-based therapies for the treatment of severe autoimmune diseases and cancer, today announced the dosing of the first patient in a Phase II proof-of-concept study of ARGX-113 in patients with primary immune thrombocytopenia (ITP).
"The initiation of the second Phase II trial for our lead candidate, ARGX-113, is an important milestone for the company as it exemplifies the breadth of potential this compound has to address a wide variety of diseases that are driven by pathogenic IgGs," commented Nicolas Leupin, CMO of argenx. "We believe ARGX-113 could be a breakthrough therapy in an indication like ITP where current therapies do not adequately address symptoms or achieve remissions."
- From argenx Press Release
"Protalex’s orphan drug PRTX-100, for the potential treatment of persistent, chronic immune thrombocytopenia (ITP) is currently in a clinical trial and has been granted orphan drug status by the FDA.
PRTX-100 is being tested in an open-label, Phase 1/2 dose escalating study in patients who have previously received treatment with a thrombopoietin receptor agonist and at least one additional ITP therapy."
- By Andrew Black, Rare Disease Report
"The FDA has approved Gammaplex 10% (immune globulin intravenous [human], 10% liquid) for the treatment of primary immunodeficiency (PI) and chronic immune thrombocytopenic purpura (ITP) in adults.
In 2013, the FDA approved Gammaplex 5% and the newer solution has an immune globulin G (IgG) concentration of 100g/L that is stabilized with glycine.
The main benefit of the increased concentration is that infusion time is significantly reduced. In a study comparing the two solutions, the mean infusion time for Gammaplex 10% was 1 hour and 51 minutes compared to 2 hours and 46 minutes with Gammaplex 5%"
- James Radke, Rare Disease Report
"...Instead of lying in a hospital bed and receiving medical care, 10-year old Bakhtawar Shafiq, who is suffering from a rare blood disorder, was forced to hold a protest demonstration in front of the Karachi Press Club (KPC) on Tuesday for a medicine that is vital for her survival but very costly and beyond the means of her poor parents.
“Doctors have diagnosed Bakhtawar with chronic immune thrombocytopenia purpura (ITP), which is a bleeding disorder in which the blood doesn’t clot as it should. They have prescribed her tablet Revolade 50mg, whose single tablet costs around Rs3,660,” said the girl’s mother after the demonstration."
By M. Waqar Bhatti. From thenews.com.pk.
Dreieich, Germany | January 25, 2017
"Biotest AG today announced that the first patient has been treated in study no. 992 - a phase III study investigating IgG Next Generation as immunomodulatory therapy in patients diagnosed with chronic primary immune thrombocytopenia, an autoimmune disease in which the immune system attacks and destroys the body's own platelets, the cells that prevent bleeding in blood vessels and facilitate clotting.
The study is planned to include approximately 40 patients and will be conducted in a total of 18 study sites in Germany, Hungary, Spain, Bulgaria, Czech Republic and Serbia."
- Source: Biotest
Today, President Obama signed into the law the 21st Century Cures Act. The massive bill will bring much needed funding to medical research, including research focused on rare diseases. Many in the rare disease community have worked for years to get this legislation passed and we at Rare Disease Report want to thank all that have helped make this dream a reality.
Some of the highlights of the 21st Century Cures Act that are of interest to the rare disease community include:
- By James Radke, PhD. RareDR.com.
The latest research was presented at the ITP Breakfast Meeting at ASH in San Diego on Saturday, December 3, 2016.
Here are some of the findings:
1. One year after diagnosis of ITP in children, most children will recover and 10% will become chronic.
2. Steroids given to children who do not have significant bleeding tend to have ITP longer showing that steroid therapy may actually delay recovery.
3. The ITP patient's condition rather than the number of platelets should determine treatment.
4. Not every child with ITP is eligible for a watch-and-wait strategy, which is a time consuming procedure.
5. Patients with ITP refractory to multiple treatments may respond to a combination of TPO and immunosuppressive agents.
6. According to three different studies, 15-53% of ITP patients went into remission after discontinuing a TPO agent.
More in-depth information is published in the December 29, 2016 edition of the PDSA E-News:
The following statement was issued by Peter L. Saltonstall, President and CEO of the National Organization for Rare Disorders (NORD), following the results of the recent U.S. election.
“Many of us were surprised by the outcome of the presidential election last week. The pollsters and the pundits were predicting a different outcome. But the long election season has ended and we have a new President, a new Congress, and a series of new challenges for NORD and the rare disease community.
Healthcare is one of the top priorities of President-elect Trump. At the top of the agenda is his pledge to replace the Affordable Care Act. With both houses of Congress having a Republican majority, the incoming President is likely to have support for his goals. The new President also has made comments about the drug development process and about the drug approval process at FDA and about other issues that may directly affect the rare disease community."
"AARDA’s #AutoimmuneHeroes campaign recognizes the work of individuals and organizations that have advanced the autoimmune disease agenda in the areas of research, patient safety, advocacy, public education and awareness, service and volunteerism, among others.
Created and facilitated by AARDA, the NCAPG and its members advocate tireless on key issues – patient safety, step therapy, access to medicine, the Affordable Care Act – that impact the 50 million Americans who suffer from one or more of the 100+ known autoimmune diseases."
- From AARDA Press Release
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