I am in an extended family caregiver circle for a child recently diagnosed with a rare disease. The journey has been humbling and heartbreaking, and my lens continues to change with the work that needs to be done in this space. It’s not just a day, week, or month to highlight; it’s all day, every day, a constant struggle for families and caregivers every hour, a deeply vulnerable space where they need to have Clinical Research as a care option, no matter where they are in the world.
I know I’m privileged to work in this industry to help make a difference, helping companies navigate the development pathways, communicating with regulatory agencies, working with my colleagues to help incorporate the patient voice, and ensuring the experiences that patients and caregivers have in a clinical trial have them front and center not just data points in studies.
My personal lens serves only to raise the game with what we need to do. I am here to help make treatments for rare diseases less rare. Patients can’t wait.
That’s a wrap (for now)!
Our goal in creating this page was to provide a forum for everyone who supports rare diseases to connect and share insights from within their community, so that we can all learn from each other. Generally speaking, industry might use LinkedIn as a communication outlet, researchers frequently use Twitter, and patients and advocacy groups often use Facebook. There is no one platform that is available to all people who are invested in rare disease research.
That is why we created Rare Moments: to bring together all the voices and inspire sharing of resources and collaboration.
Thank you to everyone who participated in this effort. We are grateful to you for sharing your thoughts with the wider rare disease community.
I had such a fulfilling experience visiting my members of Congress as part of Rare Disease Week on Capitol Hill. My fellow advocates and I raised our voices to encourage their support of policies that benefit rare disease patients. Hearing everyone’s stories was so inspiring.
We are proud to support and bring awareness to Triple Negative Breast Cancer Day which was yesterday, March 3rd. Individuals in the United States have a 1 in 8 chance of developing breast cancer and TNBC accounts for 10-25% of those breast cancers. Me and many others at Worldwide are excited to take part in the many events occurring throughout March that bring awareness to this disease. Please join us at any of the highlighted events listed below.
tnbcfoundation.org/about-us/our-events/tnbc-day/virtual-events-2024TNBC Day | Triple Negative Breast Cancer Foundation
Every day in March is TNBC Day! Triple Negative Breast Cancer Day is the only global event dedicated to raising both awareness and funds exclusively for triple negative breast cancer. Find out how you can get involved!
tnbcfoundation.org
www.linkedin.com/posts/canaryadvisorsllc_raredc2024-rarediseaseday-activity-7163158752681000960-1zXJCheck out the Minnesota Twins’ Target Field lit up for Rare Disease Day!
My nephew Steffen was born with a rare genetic disorder -CHARGE Syndrome- 22 years ago. Although he is deaf, blind, and has multiple other issues, he is not medically fragile. Steffen is nonverbal and has Autistic tendencies. Nothing holds Steffen back. Whatever the family does, so does Steffen. Whether it is whitewater rafting, skiing downhill, skating, or a dance video party, Steffen is right there. His laugh and joking nature are a joy to be around. Thanks to great health care providers and interveners/caregivers, Steffens life is full and brings joy and love to all those that know him. His parents are members of the CHARGE Syndrome foundation and provide support to new parents who receive the rare diagnosis of CHARGE. It means everything to talk to someone who has been there. Today is special day for our family. Together we can raise awareness and influence change.
The CHARGE Syndrome Foundation
A better world for people with CHARGE Syndrome.
www.chargesyndrome.orgOn this Rare Disease Day, I pause and give gratitude to all those in our community who help move the needle in researching and fighting Rare Diseases. A very close friend of mine was diagnosed with NMO (Neuromyelitis optica) several years ago and fortunately has found a medicine that has kept her in remission from relapses. She goes every two weeks for an infusion and will continue this for the rest of her life. It amazes me the resilience and positive attitude she carries with her in the face of this terrible rare disease. I love working for a passionate organization that helps companies execute Rare Disease trials in hopes of finding new treatments for these patients!
⭐ Today is #RareDiseaseDay! Join us as we #leap4rare to show our support for the 300 million people worldwide living with rare diseases. The Orphan Disease Center is dedicated to advancing research and providing support all year round. Let’s unite in solidarity and make a difference together! #raredisease #supportresearch
www.linkedin.com/posts/orphan-disease-center-university-of-pennsylvania_rarediseaseday-leap4rare-raredisease-activity-7168989114392911873-8ZZsOrphan Disease Center | University of Pennsylvania on LinkedIn: #rarediseaseday #leap4rare #raredisease #supportresearch
â Today is #RareDiseaseDay! Join us as we #leap4rare to show our support for the 300 million people worldwide living with rare diseases. The Orphan Diseaseâ¦
www.linkedin.comRead our latest blog post to learn about the cell and gene therapy trends Trinity Life Sciences is watching for 2024!
This Rare Disease Day, I am thinking about how phenomenal it is to see how rare oncology patients have benefitted from the 6 CAR T treatments now available! It is exciting to watch how continued advancements have turned CAR T into a new development paradigm for autoimmune diseases – looking forward to seeing these additional patient communities benefit from cell therapy!