Wednesday, September 22 at 5pm EST
Thursday, September 23 at 11:45am to 12:30pm EST
At Horizon, we believe science and compassion must work together to transform lives. A company focused on researching, developing and commercializing medicines that address critical needs for people impacted by rare and rheumatic diseases, we apply scientific expertise and courage to bring clinically meaningful therapies to patients.
That’s why we think differently about research and development, so we can deliver in new ways for the patients and families we serve.
We believe our responsibility also extends to the communities where we live and work, which is why we are committed to helping transform and better the lives of those who need it most.
To make a bigger difference, we nurture a work environment that reflects the diversity of our patients. We build teams who work together with speed and urgency, because each extra day it takes to get something done is an extra day that someone may not have the medicine or support they need. Because at Horizon, success is measured by the number of lives we touch, the number of lives we change and those we work tirelessly to help save.
Primary immunodeficiencies may change hematology/oncology management: Given the overlap in signs and symptoms of primary immunodeficiencies (PI) with hematology and oncology, it’s quite likely that you may have seen or will potentially see a patient who appears to have classical benign lymphopro-liferation, cytopenias or lymphoma – but actually has PI-derived lymphoproliferation, ITP, AIHA, or lymphoma.
Identifying an underlying PI may change your management for that patient. For example, outcomes in patients with undiagnosed APDS, or activated PI3Kδ syndrome, who had lymphoma were significantly worse than the norm for lymphoma in a non-PI population.
APDS is a PI with a heterogenous presentation: Patients with APDS often present with a complex history of infections. These infections range from the unremarkable, such as frequent upper respiratory infections, to the unusual, such as severe or chronic herpes. But these patients may also initially present with autoimmunity or even lymphoma.
As the understanding of PI grows, it’s clear that immune dysregulation may be the prominent feature for a patient. This dysregulation can appear in many organ systems; nearly all patients with APDS have one or more hematological symptoms. These heterogenous presentations can make diagnosis elusive.
Pharming offers sponsored no-charge genetic testing for patients you may suspect have APDS: Pharming understands that the only way to diagnose a PI is via a genetic test, but that the barriers to these tests are many. A chief goal for Pharming is to remove these barriers so your patients can get the diagnosis and appropriate care they need.
We’d hope you will stop by the booth to talk about APDS, our genetic testing program, and about how we can best serve the hem/onc community.
For more info visit:
Sobi is an international specialty healthcare company dedicated to rare diseases. Our mission is to develop and deliver innovative therapies and services to improve the lives of patients. The product portfolio is primary focused on Hemophilia, Inflammation and Genetic diseases. We also market a portfolio of specialty and rare disease products across Europe, the Middle East, North Africa and Russia for partner companies. Sobi is a pioneer in biotechnology with world-class capabilities in protein biochemistry and biologics manufacturing. More information is available at https://sobi-northamerica.com/
For more info visit:
X4 Pharmaceuticals is a late-stage biopharmaceutical company. X4 is a leader in the discovery and development of novel therapies for the treatment of rare diseases resulting from dysfunction of the CXCR4 pathway. The lead candidate, mavorixafor, a first-in-class, oral, small molecule antagonist of CXCR4. X4 believes that inhibition of CXCR4 creates potential for mavorixafor therapeutic benefit across a variety of diseases. The efficacy and safety of mavorixafor is being evaluated in a Phase 3 clinical trial in WHIM syndrome, and in two Phase 1b clinical trial – in combination with ibrutinib in Waldenström’s macroglobulinemia, and as monotherapy in Severe Congenital Neutropenia.
At Blueprint Genetics, we are proud to offer comprehensive and high-quality genetic testing for a wide variety of inborn errors of immunity. Our Comprehensive Immune and Cytopenia Panel (642 genes) is designed to detect clinically relevant, non-coding variants, and includes more than 90% of the genes listed in the 2019 International Union Immunological Societies paper (PMID: 31953710). We are also proud to offer a 298 gene Primary Immunodeficiency Panel and 135 gene Bone Marrow Failure Panel. Stay tuned for a panel update coming soon!
Advocacy Partners Thursday Only
The mission of the National Neutropenia Network is to promote awareness, education, and research, and to provide a support system for patients with severe chronic neutropenia (SCN) and their families through a national resource network.
There are 4 types of Neutropenia: Congenial, idiopathic, cyclic, and autoimmune. Congenial & cyclic are typically genetic while idiopathic and autoimmune can at any time for unknown reasons.
Individuals with neutropenia can deal with a variety of symptoms from fevers, pain, and chronic infections. Some appear healthy to others and live their lives while other suffer chronic debilitating infections where they cannot work.
Neutropenia is a rare condition, affecting males and female equally and occurring in 0.5 to 1 person per 1 million in the U.S. Many feel isolated without the connection of others with the same condition.
The National Neutropenia Network, or NNN, provides awareness, education, and support to individuals throughout the world. We provide education through our website, social media platforms, webinars, and conferences. The NNN provides awareness through social media platforms, collaboration with others such as pharmaceutical companies, NORD, SCNIR, and others. Support occurs with frequently asked questions, which doctors are in their area, peer support program, and our Junior & Senior Ambassador programs.
For more information about the National Neutropenia Network or neutropenia, please visit our website at www.neutropenianet.org.
DKMS was founded in 1991 after one family’s struggle to find a lifesaving bone marrow donor – growing from 3,000 donors to 68,000 in just the first year. Since then, the DKMS team has been dedicated to facilitating as many second chances at life as possible in order to give families more time with their loved ones. We provide individualized service and care to every single patient – sharing resources and tools, our U.S. based and international connections, and best practices for reaching and inspiring the largest number of people possible.