About Orsini
Patient Advocacy
Orsini collaborates with a diverse network of patient advocacy organization that are driving progress for individuals and families impacted by rare and complex conditions. We are proud to support their efforts to raise awareness, advance research, and provide critical resources to rare disease communities.
At Orsini, we are committed to fostering meaningful partnerships with a wide array of patient advocacy organizations that champion the needs of individuals and families affected by rare and complex medical conditions. These collaborations are at the heart of our mission to make a lasting impact in the rare disease community.
By supporting the initiatives of these organizations, we help amplify the voices of patients and caregivers, ensuring their stories are heard and their needs are addressed. Together, we are building a stronger, more informed, and more compassionate community—one that empowers patients, drives innovation, and brings hope to those living with rare and complex conditions.
Upcoming Events
HAEA Conference
7/10/25 – 7/13/25, Baltimore, MD
NNPDF Family Support & Medical Conference
7/10/25 – 7/13/25, Charlotte, NC
MPS Lurie Family Day
8/16/25 – 8/16/25, Chicago, IL
Duke Pediatric Pompe Conference
8/23/25 – 8/23/25, Virtual Event
DNPKUA San Diego Lifting the Limits for PKU and On the Horizon: One Day Community Conference
9/5/25 – 9/6/25 , San Diego, California
Jett Foundation World Duchenne Awareness Day and Gals for Cal Triathlon
9/6/25 – 9/7/25 , Quincy and Hopkinton, MA
Cure SMA Chicago Walk-n-Roll
9/21/25 – 9/21/25 , Chicago, IL
Alex Rezcalla Golf Tournament
9/29/25 – 9/29/25, Powell, OH
Amyloidosis Forum Annual Meeting
10/1/25 – 10/1/25, Location TBD
International HypoPARAthyroidism Conference
10/3/25 – 10/5/25 , Grapevine, TX
Cure SMA Raleigh Walk-n-Roll
11/8/25 – 11/8/25, Raleigh, NC
PFF Summit
11/81325 – 11/15/25, Chicago, IL
Patient Advocacy Partners
The Alex Rezcalla Foundation advances the development of therapies for individuals with Duchenne muscular dystrophy (DMD) through advocacy and research funding. It raises awareness of the DMD experience by hosting events, engaging the public, and building networks of support.
Learn more at alexrezkallafoundation.org
The Amyloidosis Research improves the lives of patients with amyloidosis and accelerates cutting edge research through collaborative, groundbreaking initiatives. ARC provides support, information, and resources to patients and caregivers, empowering them to access the best care possible.
Learn more at aci.org
Cure SMA is dedicated to finding treatments and a cure for spinal muscular atrophy (SMA) by funding research and supporting families. Since 1984, Cure SMA has grown to be the largest U.S.-based network of individuals, families, clinicians, and research scientists working together to advance SMA research, support individuals and families impacted by SMA, and educate public and professional communities about SMA.
Learn more at www.curesma.org
The US Hereditary Angioedema Association (HAEA) is a 501 (c)(3) non-profit advocacy and research organization serving people living with the rare genetic condition, Hereditary Angioedema (HAE), and their families. Founded and staffed by people with HAE and caregivers, the US HAEA is committed to actively engaging our community in a wide variety of grassroots activities that promote HAE education and awareness.
Learn more at haea.org
The HypoPARAthyroidism Association is a non-profit patient association working to improve the lives of people impacted by hypoPARAthyroidism through education, support, research, and advocacy. They raise awareness of hypoPARAthyroidism and bring change to the lives of those affected.
Learn more at hypopara.org
Jett Foundation empowers people and families impacted by Duchenne muscular dystrophy through the development of transformative programming, educational opportunities, and ongoing support for every stage of a Duchenne journey.
Learn more at jettfoundation.org
Muscular Dystrophy Association (MDA) is the #1 voluntary health organization in the United States for people living with muscular dystrophy, ALS, and over 300 other neuromuscular conditions. For 75 years, MDA has led the way in accelerating research, advancing care, and advocating support and inclusion of families living with neuromuscular disease.
Learn more at mda.org
The National MPS Society is committed to making a difference in the lives of MPS families through support, research, education, and advocacy. Families from around the world gain a better understanding of these rare genetically determined diseases through the Society’s assistance in linking them with healthcare professionals, researchers, and, perhaps most importantly, each other.
Learn more at mpssociety.org
The National Niemann-Pick Disease Foundation, Inc. (NNPDF) is a non-profit, patient advocacy and family support organization dedicated to supporting and empowering patients and families affected by Niemann-Pick disease, through education, collaboration and research. Founded in 1993, NNPDF serves families throughout the nation at all stages of their Niemann-Pick journey.
Learn more at nnpdf.org
The National PKU Alliance (NPKUA) works to improves the lives of individuals with PKU, pursue a cure by expanding research, and provide education and support to individuals living with PKU and their caregivers. Since its inception in 2008, NPKUA has supported numerous successful fundraising events, conferences, and internal support programs and has remained steadfast in its support of research endeavors.
Learn more at npkua.org
Parent Project Muscular Dystrophy (PPMD) is the largest nonprofit organization in the United States focused entirely on Duchenne muscular dystrophy. They take a comprehensive approach in the fight against Duchenne—funding research, raising awareness, promoting advocacy, connecting the community, and broadening treatment options.
Learn more at parentprojectmd.org
The Pompe Alliance is a Public Services Organization dedicated to the ideas of support, empowerment, and progress. It seeks to provide supportive services and information to patients, caregivers, medical professionals, and community stakeholders through a number of initiatives: financial assistance, advocacy, education, mentoring, and research recruitment
Learn more at pompealliance.com
The mission of the Pulmonary Fibrosis Foundation is to accelerate the development of new treatments and ultimately a cure for pulmonary fibrosis. Until this goal is achieved, the PFF is committed to advancing improved care of patients with PF and providing unequaled support and education resources for patients, caregivers, family members, and health care providers.
Learn more at pulmonaryfibrosis.org
The United Pompe Foundation was formed to assist patients and/or their families with medical costs and other expenses that these patents and families face and may not be able to cover, or fully cover, through their insurance.
Learn more at unitedpompe.com