Partners
Meet Our Partners
We secure partners who are working towards our mutual goal of finding a treatment or cure for hypoPARAthyroidism. We also partner with organizations that can help us educate both patients and physicians about our disease. Finally, we partner with organizations that can help us help patients with navigating insurance, disability, and other treatment needs.
Alexion, AstraZeneca Rare Disease
Alexion, AstraZeneca Rare Disease is focused on serving patients and families affected by rare diseases and devastating conditions through the discovery, development and delivery of life-changing medicines. A pioneering leader in rare disease for more than three decades, Alexion was the first to translate the complex biology of the complement system into transformative medicines, and today it continues to build a diversified pipeline across disease areas with significant unmet need, using an array of innovative modalities. As part of AstraZeneca, Alexion is continually expanding its global geographic footprint to serve more rare disease patients around the world. It is headquartered in Boston, US.
TO LEARN MORE ABOUT Alexion, AstraZeneca Rare Disease
VISIT THEIR PARTNER PAGE Alexion, AstraZeneca Rare Disease
Ascendis Pharma
Ascendis Pharma is applying its innovative technology platform to build a leading, fully integrated biopharma company focused on making a meaningful difference in patients’ lives. Guided by our core values of patients, science, and passion, we use our TransCon® technology platform to create new and potentially best-in-class therapies.
TO LEARN MORE ABOUT ASCENDIS VISIT THEIR PARTNER PAGE ASCENDIS
Bridgebio/Calcilytix
Calcilytix Therapeutics is a clinical-stage biopharmaceutical company based in California, USA. We are an affiliate of BridgeBio Pharma, whose goal is to create life-altering medicines that target well-characterized genetic diseases at their source.
TO LEARN MORE ABOUT BRIDGEBIO VISIT THEIR PARTNER PAGE BRIDGEBIO/CALCILYTIX
Global Genes
Global Genes is a 501(c)(3) non-profit organization dedicated to eliminating the burdens and challenges of rare diseases for patients and families globally.
Global Genes’ story began with our loved ones. We are the friends, family and supporters of patients close to us who are affected by rare disease. We understand the confusion, the overwhelming experience of the unknown and the feelings of isolation all too well. And we know we aren’t alone. That’s the reason Global Genes was born – to connect, empower and inspire the rare disease community.
Provide patient advocates with a continuum of services to accelerate their path from early support and awareness through research readiness, using a collaborative approach that involves biopharma, researchers and funders, with data as a central core.
We are also apart of the Global Genes Global Advocacy Alliance
Follow Global Genes on Facebook, Instagram, YouTube and Twitter
MBX Biosciences
MBX Biosciences is pioneering Precision Endocrine Peptide™ (PEP™) therapeutics to help people with endocrine disorders live fuller and healthier lives. Our mission is to restore health, simplify management and enable greater access for patients with endocrine diseases. The company’s lead product candidate for the treatment of hypoPARAthyroidism is currently in development and is designed to restore PTH physiology and correct the mineral imbalances to alleviate symptoms of the disorder. MBX is based in Carmel, Indiana. To learn more, please visit the company website at www.mbxbio.com and follow on LinkedIn and Twitter.
TO LEARN MORE ABOUT MBX BIOSCIENCES VISIT THEIR PARTNER PAGE MBX BIOSCIENCES
NORD
NORD (National Organization for Rare Diseases) advances practical, meaningful, and enduring change so people with rare diseases can live their fullest and best lives. Every day, we elevate care, advance research, and drive policy in a purposeful and holistic manner to lift up the rare disease community.
Follow NORD on Facebook, Instagram, LinkedIn, Twitter and YouTube
Or contact NORD here
Orsini Rare Disease Pharmacy Solutions
Orsini | Rare Disease Pharmacy Solutions
Orsini is proud to be a specialty pharmacy for Yorvipath, an FDA-approved parathyroid hormone analog indicated for the treatment of hypoparathyroidism in adults. A leader in pharmacy solutions for rare diseases and complex therapies, we have been providing patients with compassionate care since 1987. We are built to support rare disease patients and their families and to simplify how they access revolutionary treatments. Our mission is simple: Ensuring that No Patient is Left Behind™. Please reach out to us if you would like to access Yorvipath through Orsini, or if you have any questions or concerns at any time – we’re here to help!
Visit us online to learn more about Orsini:
HPA Partner Page | Endocrinology Center of Excellence | LinkedIn | Facebook | YouTube
PantherRx
PantherRx
As The Rare Disease Pharmacy® , PANTHERx uniquely serves the needs of our patients and their caregivers, pharmaceutical manufacturer partners, prescribers, and payers.
Check out their website here
Septerna
Our mission is to discover, develop, and commercialize medicines that improve the lives of patients by combining the potential of high-value GPCR drug targets with the power of state-of-the-art small molecule drug discovery technologies. Septerna’s Native Complex Platform™ is the engine that will make this happen, moving ideas for disease-modifying therapies from possibility to reality.
TO LEARN MORE ABOUT SEPTERNA VISIT THEIR PARTNER PAGE SEPTERNA
Takeda Pharmaceutical Company
Takeda is a patient-focused, values-based, R&D-driven global biopharmaceutical company committed to bringing Better Health and a Brighter Future to people worldwide. Our passion and pursuit of potentially life-changing treatments for patients are deeply rooted in over 240 years of distinguished history in Japan.
Our corporate philosophy tells the story of Takeda — who we are, what we do, how we do it, and why it matters. It reminds us how together, we can achieve “better health for people, brighter future for the world”.
We connect to our history and Japanese heritage through everything we do today, to bring our purpose, values, vision, and imperatives to life.
TO LEARN MORE ABOUT TAKEDA VISIT THEIR PARTNER PAGE TAKEDA