Thank you.

Thank you to our Industry, Advocacy and Philanthropic partners for your support towards the 2022 NICER symposium.

To learn more please visit:

https://www.barthsyndrome.org/

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

Race For Immunology support the education and research around rare immunologic disorders to increase early diagnoses and save lives. To learn more please visit: www.raceforimmunology.org

Neutropenia is a very rare blood condition that causes a reduced number or complete lack of neutrophils. To learn more please visit: https://neutropenianet.org/

The Cohen Syndrome Association was founded by parents to raise awareness of this disease with the goal of educating parents and professionals to assure earlier diagnosis and medical interventions. To learn more please visit: www.cohensyndrome.org

x4 Pharmaceuticals

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 Pharmaceuticals

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

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
www.novartis.com.

ADMA Biologics

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 gwetzstein@admabio.com or Rickey Miller rmiller@admabio.com

https://www.admabiologics.com/ https://www.asceniv.com/

Sobi Pharmaceuticals

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.

www.sobi-northamerica.com
Video link: https://www.youtube.com/watch?v=BZtAI7qxiHs

Horizon Therapeutics

We are Horizon Therapeutics, a global biotechnology company driven by a simple idea. We believe that science and compassions must work together to transform lives. horizontherapeutics.com
www.actimmune.com

Pharming

A global biopharmaceutical company dedicated to transforming the lives of patients with rare, debilitating, and life-threatening diseases.Home | Pharming Group N.V.