Thank you to our Industry, Advocacy and Philanthropic partners for your support towards the 2022 NICER symposium.
To learn more please visit:
Barth Syndrome Registry https://qrco.de/bthsbrr
Barth Syndrome Foundation Research https://qrco.de/bthsresearch
Tools For School https://qrco.de/tools4school
Care Management Toolbox https://qrco.de/bthscare
Developing novel therapeutics to treat people with chronic neutropenic disorders
X4 Pharmaceuticals is a late-stage clinical biopharmaceutical company and a leader in the discovery and development of novel therapies for the treatment of diseases of the immune system via antagonism of the CXCR4 pathway, with a focus on rare diseases and those with limited treatment options. We believe that inhibition of the CXCR4 receptor creates the potential to provide therapeutic benefit across a wide variety of diseases, including chronic neutropenic disorders and certain types of cancer.
Agios is a biopharmaceutical company that is fueled by connections. The Agios team cultivates strong bonds with patient communities, healthcare professionals, partners and colleagues to discover, develop and deliver therapies for genetically defined diseases. These diseases include Pyruvate Kinase Deficiency, Alpha- and Beta-Thalassemia, and Sickle Cell.
Novartis is reimagining medicine to improve and extend people’s lives. As a leading global medicines company, we use innovative science and digital technologies to create transformative treatments in areas of great medical need. In our quest to find new medicines, we consistently rank among the world’s top companies investing in research and development. Novartis products reach more than 800 million
people globally and we are finding innovative ways to expand access to our latest treatments. About 109,000 people of more than 145 nationalities work at Novartis around the world. Find out more at
ADMA Biologics is an end-to-end commercial biopharmaceutical company committed to manufacturing, marketing, and developing specialty plasma-derived products for the prevention and treatment of infectious diseases in the immune compromised and other patients at risk for infection. Our devotion to these underserved populations fuels us, and our hands-on approach to production and development sets us apart. Contact: Gene Wetzstein firstname.lastname@example.org or Rickey Miller email@example.com
As the North American affiliate of biopharmaceutical company Sobi™, our vision is to provide sustainable access to innovative therapies and transform the lives of people affected by rare diseases. Our product portfolio is focused on immunology and genetics/metabolism.