PROS Foundation -Pathways for Rare and Orphan Studies

We are proud to be a Gold sponsor of the 2023 WOD Congress, the world’s most important orphan drug and rare disease event! We are a member of Global Genes and its RARE Corporate Alliance, a partnership of rare disease industry stakeholders committed to forging and supporting collaboration between industry and patient communities. We are proud to showcase the work of Kam Redlawsk on our walls at H4B Boston.

Through collaborations with world-renowned academics and clinicians, we are moving science forward to develop life-saving gene therapies for rare and ultra-rare disease patients overlooked and underserved by traditional drug development efforts. Our collaborative framework reimagines the drug development process to provide an accelerated pathway for the development of life-saving therapeutics that previously did not exist for people with rare and ultra-rare neuromuscular diseases. Moreover, our model offers industry collaborators a mechanism to expand their drug development pipeline. As the scope of our research and development grows, so does our need to expand our network of collaborators to advance therapeutics for rare and ultra-rare neuromuscular diseases, speed drug development for orphan diseases, and expand access to treatments to broader populations.

We are a dedicated team of physicians, nurses, scientific researchers, administrative staff, and patient advocates working to provide comprehensive medical care, integrate science and clinical medicine, and share insights to improve the health of children and adults facing rare genetic disorders. From our humble beginnings, we have provided a medical home for children and adults with rare genetic conditions. For over 35 years, we have been steadfast in our mission of providing efficient and affordable healthcare to vulnerable individuals with genetic diseases. Our collaborations help us provide affordable specialty care on-site at the Clinic, making services more accessible to families. Our clinic serves as a trusted medical home for families working to prevent and treat genetic illness in their children. Our clinic serves as a trusted medical practice for children and adults facing rare genetic disorders. Inside is filled with cutting-edge gene sequencing tools that allow us to deliver highly personalized care—a precise treatment option for the right patient at the right time.

OUR MISSION is to help rare disease patients by ending their diagnostic odyssey. Our research platform will let you and your provider look up all the known information about your diagnosed disease in minutes - related genes, repurposed drugs, existing care, and active clinical trials. Our platform is built to help you navigate the most challenging cases.

The devastating impact of COVID-19 on the general population is well-documented—but less is known about the millions of people living with rare diseases. Our network, now in its fourth five-year funding cycle, is a partnership with funding and programmatic support provided by Institutes, Centers and Offices across NIH.

At RareDisease.net we empower patients and caregivers to take control of rare diseases by providing a platform to learn, educate, and connect with peers and healthcare professionals. RareDisease.net can have an enormous impact on the lives of patients and their loved ones. At RareDisease.net, we strive to provide you with the most accurate and relevant information. RareDisease.net also features articles contributed by Physicians, Patient Advocates or other Healthcare Experts. RareDisease.net does not edit these articles, as they represent the thoughts, opinions, and judgment of their authors. RareDisease.net accepts responsible advertising and sponsorships, however sponsors do not influence editorial content in any way. RareDisease.net is brought to you by Health Union, LLC, which does not manufacture, distribute, market or sell any product to cure, diagnose or prevent rare diseases or any health condition. Although there is no cure for rare diseases, we hope the information provided by patients, caregivers, and healthcare professionals at RareDisease.net will help improve your quality of life.