How Rare Diseases Became Not So Rare at RMH-NY
For decades, Ronald McDonald House New York (RMH-NY) has provided comfort, care, and support to families navigating their child’s serious illness. While pediatric cancer continues to be the primary focus, RMH-NY recognized that many families facing rare diseases needed the same level of assistance. Over time, the organization expanded its reach to ensure that these families—often feeling isolated by the rarity of their child’s condition—could also find a place of respite and community.
As Rare Disease Day approaches on February 28th, it is an opportunity to reflect on RMH-NY’s evolving mission and the vital role this day plays in raising awareness for the millions affected by rare diseases.
A Deliberate and Transformative Expansion
The decision to welcome families affected by rare diseases was not made overnight. While RMH-NY has long accommodated some exceptions beyond pediatric cancer, medical professionals increasingly sought housing for families needing specialized treatments beyond oncology. Recognizing this growing demand, RMH-NY officially expanded its mission, a shift made possible by dedicated staff, volunteers, and leadership.
This strategic move ensured that children battling rare diseases could access the same compassionate support RMH-NY had long provided to pediatric cancer patients.
A Pandemic-Era Plan for Growth
The COVID-19 pandemic presented challenges for RMH-NY, particularly in restoring pre-pandemic capacity. While the House never closed its doors, volunteer engagement declined, and families were initially hesitant to return to communal spaces. Yet, leadership recognized an opportunity not just to rebuild, but to grow.
During this period, RMH-NY developed a strategic plan to expand its impact beyond its 73rd Street location. This plan led to the creation of additional Family Rooms in hospitals and the expansion of the Community Health team. These initiatives allowed RMH-NY to serve more families than ever before in its 45-year history.
A key element of this growth was further extending services to children with rare diseases. This effort culminated in a brand refresh in 2021 in collaboration with Weber Shandwick led by Sharon Carter Jones, Chief Strategy, Branding, Compliance & Partnerships Officer.
Solidifying Rare Diseases in RMH-NY’s Mission
As part of this brand evolution, RMH-NY officially incorporated the phrase “and other complex illnesses” into its mission statement. This change reflected a reality that had existed for years—families seeking specialized care in New York City for rare diseases had long been welcomed at RMH-NY. Now, the organization’s messaging fully acknowledged this essential part of its mission.
Addressing the Growing Need for Local Support
Beyond serving families traveling from across the country and the world, RMH-NY also recognized the challenges faced by New York City residents. Many local families, even those living within a 50-mile radius, struggle with long and costly daily commutes to medical appointments, often spending hours in transit. Additionally, for families in multi-generational households, a lack of privacy and adequate rest spaces made caregiving even more difficult.
By expanding its care model, RMH-NY has provided housing to a more diverse group of families, offering a space where they can find both practical support and emotional connection. For many facing rare diseases, staying at RMH-NY is the first time they meet others who truly understand their journey—an invaluable sense of community in an otherwise isolating experience.
In addition to providing housing and support, RMH-NY plays a role in raising awareness about rare diseases. Celebrating Rare Disease Day highlights the strength of these children and their families while advocating for continued research and medical advancements.
Rare Disease Day: Raising Awareness for Rare Conditions
Rare Disease Day, held every year on the final day of February, serves as a platform to raise awareness for the approximately 300 million people worldwide who live with rare diseases including those living with rare forms of cancer for those currently staying in our East 73rd street House. This day is vital in educating the public about the struggles of rare disease patients and their families, while also advocating for greater recognition, funding, and research to address these conditions.
The origins of Rare Disease Day can be traced back to 2008, when it was first launched by the European Organization for Rare Diseases (EURORDIS) in collaboration with patient organizations around the world. The idea was simple yet profound: to shine a light on the millions of people affected by rare diseases, many of whom are often overlooked by the general healthcare system. The date was chosen to be the rarest day of the year, February 29th, with February 28th used in non-leap years. Since its inception, Rare Disease Day has grown into a global movement, with thousands of events organized worldwide. People wear zebra stripes, the symbol of rare disease awareness, to show solidarity with those affected.
Rare Disease Day is not just about raising awareness; it’s also about fostering action. The National Organization for Rare Disorders (NORD) has played a crucial role in advocating for equitable health policies for patients with rare diseases. NORD encourages people to join their Rare Action Network®, which allows advocates to connect with others, stay informed about key legislation, and take action to influence health policies on behalf of rare disease patients.
Stories of Families Affected by Rare Diseases at RMH-NY
At RMH-NY, families dealing with rare diseases have found not only a place to stay but a community that understands their struggles. Let’s take a closer look at the stories of some of these families and the rare diseases that have changed their lives.
Nirvan: Living with PGM3 Deficiency
Nirvan was diagnosed with PGM3 Deficiency, a rare genetic disorder that affects the immune system. This condition leaves Nirvan’s immune system unable to function properly, making him highly susceptible to infections. His family found solace at RMH-NY, where they met other families dealing with similarly rare conditions. "The House has been a lifeline," Nirvan’s mother said. "Not only did we get the medical care Nirvan needed, but we also met families who understood what we were going through. It made us feel less alone in our journey."
Read more about Nirvan and his parents and their journey to Ronald McDonald House New York.
How would you explain PGM3 to a 5th grader?
Imagine your immune system is like a superhero team protecting your body from germs. But with PGM3 Deficiency, one of the team’s most important tools—called sugars—doesn’t work right. These special sugars help immune cells talk to each other and fight off bad guys like bacteria and viruses. Without them, the superhero team gets confused, making it harder to fight infections. This can cause rashes, allergies, and make someone sick more often. Doctors help by giving medicine and keeping a close eye, like giving superheroes better gadgets to keep up the fight! 🦸♂️🦸♀️💥
Luna: Battling Hypoplastic Right Heart Syndrome
Luna was born with Hypoplastic Right Heart Syndrome (HRHS), a rare congenital heart defect that leaves the right side of her heart underdeveloped. Her parents, already exhausted by the constant hospital visits and surgeries, turned to RMH-NY for respite. Luna's family lived just outside of New York City in New Jersey and the hours of commuting on a daily basis were not working. Luna's mom juggled caring for all of Luna's brothers and sisters while living in a multi-generational home with constant demands on her time. Finding a place to rest, that was close to the hospital and quiet
How to explain Hypoplastic Right Heart Syndrome to a 5th grader.
Imagine your heart is like a two-pump system—one side sends blood to the lungs to get oxygen, and the other side sends that oxygen-filled blood to the rest of your body. But with Hypoplastic Right Heart Syndrome (HRHS), the right side of the heart is too small or not working well—kind of like a broken water pump that can’t send enough water where it needs to go! This makes it hard for blood to reach the lungs, so kids with HRHS might get tired easily or look a little blue. But doctors can help by doing special surgeries to reroute the blood, like setting up a new system of pipes to keep everything flowing just right! ❤️🚰
Francis: Diagnosed with IPEX Syndrome
Francis, a toddler, was diagnosed with IPEX syndrome, a rare genetic disorder that affects the immune system and leads to a variety of life-threatening complications. For his grandmother, Theresa, this meant months of hospital visits, surgeries, and ongoing medical care as Francis battled severe gastrointestinal issues, diabetes, and more. At RMH-NY, they found not only a place to stay but a community of people who understood the challenges they faced. "The House has been more than just a place to sleep," Theresa said. "It’s given me the strength to keep going. The families we’ve met here—facing similar challenges—have become part of our extended support system. We’re not alone in this fight, and that’s what RMH-NY provides."
Learn more about Francis and his grandmother, Theresa and their journey to RMH-NY.
How to explain IPEX Syndrome to a 5th grader.
Imagine your brain is the boss of a superhero team, giving orders to help you move, talk, and think. Now, at the very top of your brain—called the apex—there’s a super important control center. If it gets hurt (like from an injury or illness), the messages get scrambled! That means a person might have trouble moving, speaking, or recognizing things—kind of like a superhero forgetting how to fly or mix up their laser beams. Luckily, doctors and therapists are like brain trainers, helping get those superpowers back in action!
Willow: Single Anomalous Coronary Artery from the Pulmonary Artery (SACAPA)
Willow is the third known case in history of Single Anomalous Coronary Artery from the Pulmonary Artery. When Kanesha, her mother, was six months pregnant Willow was diagnosed with Tetrolagy of Fellot (another rare disease) and the doctors said she has a left diaphragmatic hernia as well. It was only eight days after her birth that they learned of the SACAPA diagnosis. Kanesha and Sam, her parents, immediately packed up their lives in Vermont to move to the city to secure Willow the specialized treatment she needed. They have been in the House for six months now.
How to explain Single Anomalous Coronary Artery from the Pulmonary Artery to a 5th grader.
Imagine your heart is a city, and oxygen is the food it needs to keep running. Normally, special highways (coronary arteries) bring oxygen straight from a power station (the aorta). But with SACAPA, one highway is connected to the wrong place, bringing oxygen-poor blood instead—like a delivery truck full of empty boxes! This makes the heart weak and tired. Doctors use cool machines to find the problem, and surgery helps move the highway to the right spot. Once fixed, the heart gets the oxygen it needs, and the person can be active and healthy again!
RMH-NY’s Role in the Rare Disease Community
For families dealing with rare diseases, RMH-NY offers more than just a place to stay. The House provides a safe place where families can rest, recharge, and connect with others facing similar challenges. The sense of community that RMH-NY offers is invaluable, especially for families who have never met anyone else with their child’s rare disease. The opportunity to share experiences, offer support, and find comfort in one another’s company is one of the greatest gifts that RMH-NY provides.
As Rare Disease Day approaches, RMH-NY continues to raise awareness and support for families of children with rare conditions. The expansion of services to include families of children with rare diseases underscores the organization’s commitment to ensuring that every child, no matter how rare their condition, has access to the support they need to thrive.
In the coming years, RMH-NY plans to continue expanding its services to reach even more families affected by rare diseases. By embracing these families and their unique needs, RMH-NY is stepping beyond the cancer diagnosis, offering a holistic approach to care that reflects the diversity of the pediatric patient population.
How You Can Get Involved
On Rare Disease Day, you can show your support for families affected by rare diseases by participating in local events, sharing stories on social media, or donating to organizations like RMH-NY and NORD. Wearing zebra stripes is a simple but meaningful way to stand in solidarity with those affected by rare diseases, and your support can make a significant impact on the lives of families in need.
As we celebrate Rare Disease Day, with a Zebra build-a-bear station at dinner on the 26th (Thank you, Bloomberg and RCANO!) remember that although these diseases are rare, the need for support is universal. Together, we can make sure that no family feels alone in their journey.
