Learn about health issues and treatments for children with spina bifida.

Original title: “Infants, Toddlers, and Children”

One in 10 Americans lives with a rare disease. Whether it’s a family member, friend, coworkers, classmate, neighbor, or even yourself, rare disease may touch your life, as it does the more than 30 million Americans living with a rare disease. Yet only 5% of the roughly 10,000 known rare diseases ... One in 10 Americans lives with a rare disease. Whether it’s a family member, friend, coworkers, classmate, neighbor, or even yourself, rare disease may touch your life, as it does the more than 30 million Americans living with a rare disease. Yet only 5% of the roughly 10,000 known rare diseases have an FDA-approved therapy. Most rare disease therapies are developed by small companies, either precommercial or with just a handful of therapies on the market. Often, a single clinical program represents a company’s entire portfolio, and one setback can jeopardize a company’s survival. Home » Rare Diseases » Protecting Innovation for Rare Disease Treatments for Patients and Families in Need ... One in 10 Americans lives with a rare disease. Whether it’s a family member, friend, colleague, classmate, neighbor, or even you, rare disease may touch your life. Yet only 5% of the roughly 10,000 known rare diseases have an FDA-approved therapy. Together we have delivered over 100 treatments to patients, with hundreds more in development. This progress is driven by scientific advancements spurred by smart, tailored policy. Before passage of the Orphan Drug Act of 1982, only 10 rare disease therapies were FDA-approved.

Original title: “Protecting Innovation for Rare Disease Treatments for Patients and Families in Need - Future of Personal Health”

NORWELL, Mass., and MIAMI, Fla. (March 12, 2026) – The National Organization for Rare Disorders (NORD®) and OpenEvidence today announced a new partnership to expand access to trusted, expert-reviewed rare … Continue reading "National Organization for Rare Disorders and OpenEvidence Partner to Bring AI-Powered Rare Disease Resources to Clinicians and Patients Worldwide"

Original title: “National Organization for Rare Disorders and OpenEvidence Partner to Bring AI-Powered Rare Disease Resources to Clinicians and Patients Worldwide”

Andelyn Biosciences has announced the launch of The Andelyn Foundation, a nonprofit organization created to support families living with rare and ultra-rare diseases through awareness… Andelyn Biosciences has launched The Andelyn Foundation, a nonprofit organization focused on supporting families affected by rare and ultra-rare diseases through awareness and fundraising. The foundation’s community initiatives include the annual “Fore One Purpose” golf tournament, which has donated $100,000 to patient-founded organizations. The foundation will focus on two priorities: supporting families and raising awareness of rare diseases. Each year, the organization plans to direct funds to a selected patient-founded group to support activities such as research advancement, travel, clinical readiness, or urgent care needs. The foundation aims to address challenges faced by families affected by rare diseases. More than 10,000 rare diseases have been identified worldwide, affecting about 1 in 10 Americans. However, 95% of these diseases still do not have an FDA-approved treatment, according to the company. “Our work has always been guided by the belief that science and compassion go hand in hand. The Andelyn Foundation allows us to live that belief in a different way by exposing the human stories behind the science.” Wade Macedone, CEO of Andelyn Biosciences and member of The Andelyn Foundation Board

Original title: “Andelyn Biosciences Launches The Andelyn Foundation to Support Rare Disease Families - PharmaSource”

From February 24-26, 2026, the EveryLife Foundation for Rare Diseases (EveryLife Foundation) convened hundreds of patients, caregivers, advocates, researchers, and policymakers from 49 states, the District of Columbia, and Puerto Rico for […] The post EveryLife Foundation for Rare Diseases Emboldens Advocates and Urges Congress to Prioritize Patients During 15th Annual Rare Disease Week on Capitol Hill appeared first on EveryLife Foundation for Rare Diseases.

Original title: “EveryLife Foundation for Rare Diseases Emboldens Advocates and Urges Congress to Prioritize Patients During 15th Annual Rare Disease Week on Capitol Hill”

COLUMBUS, Ohio, March 10, 2026 ... Inc., a patient-focused cell and gene therapy Contract Development and Manufacturing Organization (CDMO), today announced the unveiling of The Andelyn Foundation, a nonprofit organization dedicated to supporting families living with rare and ultra-rare diseases through awareness, ... COLUMBUS, Ohio, March 10, 2026 /PRNewswire/ -- Andelyn Biosciences Inc., a patient-focused cell and gene therapy Contract Development and Manufacturing Organization (CDMO), today announced the unveiling of The Andelyn Foundation, a nonprofit organization dedicated to supporting families living with rare and ultra-rare diseases through awareness, fundraising, and compassionate community engagement. Supporting Families Each year, the Foundation directs proceeds to a selected patient-founded organization, providing meaningful financial support where it matters most—whether for research advancement, travel, clinical readiness, or urgent care needs. Raising Awareness Through storytelling and community engagement, the Foundation works to ensure that rare diseases are seen, understood, and supported, helping "rare" feel less isolating. The Andelyn Foundation is a registered 501(c)(3) nonprofit organization established by Andelyn Biosciences to support families living with rare and ultra-rare diseases through fundraising, awareness, and community engagement. Focusing on uplifting one patient-founded organization each year, the Foundation brings people together to provide meaningful financial support and amplify stories that deserve to be seen and heard. "The Andelyn Foundation allows us to live that belief in a different way by exposing the human stories behind the science. We are committed to raising awareness for the rare disease community, amplifying family voices, and offering tangible support so that no family feels alone." "Behind every therapy in development is a family living that journey every day," said Julie Kilbarger, Andelyn Foundation Board Member and mother of Andrew, part of Andelyn's namesake, whose life was forever changed by a diagnosis of Duchenne muscular dystrophy (DMD) in early childhood.

Original title: “Andelyn Biosciences Establishes The Andelyn Foundation to Support Families Living with Rare and Ultra-Rare Diseases - BioSpace”

The week's advocacy efforts were further amplified during a Senate Special Committee on Aging hearing, which spotlighted the caregiving and healthcare challenges faced by families managing complex medical conditions, including rare diseases. With a room full of rare disease patients and families in attendance, the hearing included remarks from Senators and expert witnesses, including the EveryLife Foundation... The week's advocacy efforts were further amplified during a Senate Special Committee on Aging hearing, which spotlighted the caregiving and healthcare challenges faced by families managing complex medical conditions, including rare diseases. With a room full of rare disease patients and families in attendance, the hearing included remarks from Senators and expert witnesses, including the EveryLife Foundation's Chief Mission Officer, Annie Kennedy. By aligning grassroots' advocacy with formal congressional briefings and committee proceedings, Rare Disease Week translated patient stories into policy dialogue at the highest levels of government. "Rare Disease Week continues to show the power of coordinated advocacy in our community," said Michael Pearlmutter, Chief Executive Officer of the EveryLife Foundation for Rare Diseases. WASHINGTON, March 10, 2026 /PRNewswire/ -- From February 24-26, 2026, the EveryLife Foundation for Rare Diseases (EveryLife Foundation) convened hundreds of patients, caregivers, advocates, researchers, and policymakers from 49 states, the District of Columbia, and Puerto Rico for the 15th annual Rare Disease Week on Capitol Hill. "When patients, caregivers, researchers, and policymakers come together, we move closer to a system that recognizes the unique realities of rare diseases and delivers meaningful solutions for the entire community." While this year marked the 15th anniversary of Rare Disease Week, the EveryLife Foundation emphasized that advocacy does not end when advocates leave Washington.

Original title: “EveryLife Foundation for Rare Diseases Emboldens Advocates and Urges Congress to Prioritize Patients During 15th Annual Rare Disease Week on Capitol Hill”

Learn about the Million Hearts® goal to save 1 million lives.

Original title: “Million Hearts<SUP>®</SUP>”

Individuals living with a rare disease took turns sharing their personal experiences and health stories at a recent Rare Disease Day event held at The University of New Mexico Center for Development & Disability (CDD) in Albuquerque, NM. The gathering helped to raise awareness, facilitate ... Individuals living with a rare disease took turns sharing their personal experiences and health stories at a recent Rare Disease Day event held at The University of New Mexico Center for Development & Disability (CDD) in Albuquerque, NM. The gathering helped to raise awareness, facilitate networking, encourage problem solving, support advocacy efforts and build community around rare diseases—a reality faced by more New Mexicans than many may realize. You could hear the confidence in their voices. Sometimes it blended with shock, uncertainty, anger, pain, disbelief, frustration or even humor. But they spoke up for themselves—and others like them—confidently. These were speakers all living with a rare... These were speakers all living with a rare disease, and they took turns sharing their personal experiences and health stories at a recent Rare Disease Day event held at The University of New Mexico Center for Development & Disability (CDD). The gathering helped to raise awareness, facilitate networking, encourage problem solving, support advocacy efforts and build community around rare diseases—a reality faced by more New Mexicans than many may realize. Further, Moriarta thinks individuals with rare diseases, including New Mexicans, would benefit from more efficient and effective delivery of health care—which can hopefully be achieved through more awareness and advocacy.

Original title: “Rare Disease Day at UNM Builds Community Around a Common Human Experience | UNM HSC Newsroom”

According to the National Organization ... rare diseases identified, less than 5% have approved treatments available.2 Additionally, patients suffering from conditions that may not necessarily be considered rare — including many cancers — may either have no approved treatment options available or may have exhausted all approved treatment options. For both groups, participating in a clinical trial may be their only hope of therapy for their ... According to the National Organization for Rare Disorders (NORD), an estimated 10% of all Americans — or approximately 30 million people — half of whom are children, are affected by rare diseases.1 Of the more than 10,000 rare diseases identified, less than 5% have approved treatments available.2 Additionally, patients suffering from conditions that may not necessarily be considered rare — including many cancers — may either have no approved treatment options available or may have exhausted all approved treatment options. For both groups, participating in a clinical trial may be their only hope of therapy for their disease. Continued honesty and transparency about the investigational nature and inherent uncertainty of clinical trial treatment outcomes will ameliorate the risks of providing false hope and therapeutic misconceptions to an understandably desperate group of potential participants. Since AI tools have the potential to review medical records and clinical trial protocols more quickly and efficiently than humans, these tools will decrease the eligibility-screening time, speeding up the enrollment process and enabling participants to begin the clinical trial sooner. The use of these tools will be a boon to patients with rare diseases and those with no approved treatments. There is a common expectation that if we or our family members become ill, our doctor will be able to treat us. For Americans suffering from a rare disease, that is often not the case. However, with careful consideration in building the AI tool to enable screening for multiple trials at once and with thorough review and oversight by IRBs and Research Privacy Boards to ensure participants' privacy and the confidentiality of their records is maintained, the use of AI tools in matching potential participants to clinical trials could revolutionize the industry. ... 1 National Organization for Rare Disorders, Rare Disease Facts and Statistics, https://rarediseases.org/understanding-rare-disease/rare-disease-facts-and-statistics/

Original title: “AI Can Help With Clinical Trial Matching - New Technology - United States”

On Rare Disease Day, discover why visibility, cutting-edge science, and patient advocacy are vital for healthcare innovation and global health equity.Sinónimos de la frase clave: Rare Disease Awareness Day, February 28, World Rare Disease Day On this Rare Disease Day, visibility cannot remain mere rhetoric: it must translate into public policy, innovation, and real access. Transforming this scenario depends on an unnegotiable tripod: cutting-edge science, strategic visibility, and strengthened advocacy led by patient associations. Those living with a rare disease face a double burden: that of the pathology itself and that of social misunderstanding. Historically, patient associations were perceived mainly as emotional support networks. Today, they have consolidated as key actors in political and scientific transformation. Contemporary advocacy is not limited to demanding answers: it participates in building solutions. The journey is usually marked by the so-called “diagnostic odyssey”: on average, a patient takes between five and seven years to obtain a clinical confirmation after consulting about eight different specialists (RARE Diseases International, 2024). This delay is not harmless: it allows the progression of irreversible damage and generates profound wear: in the patient’s health, the emotional and economic balance of the family, and the healthcare system. The socioeconomic and family impact is equally significant. A study by the EveryLife Foundation estimated that the total cost of rare diseases in the United States reached nearly one trillion dollars in a single year, considering both direct costs and productivity loss. Given limited public resources, qualified advocacy participates in designing innovative financing models such as pay-for-performance agreements (risk-sharing), which seek to reconcile the incorporation of disruptive technologies with the financial stability and long-term viability of healthcare systems. Looking toward rare diseases must become a call for collective action.

Original title: “Rare Disease Day: Innovation, Equity, and Healthcare Challenges”

This report describes Kaposi sarcoma in an organ transplant recipient.

Original title: “Kaposi Sarcoma–Associated Herpesvirus Infection and Complications Among Solid Organ Transplant Recipients — United States, January 2021–September 2025”

This report describes cases of congenital rubella in Florida.

Original title: “Notes from the Field: Congenital Rubella Syndrome — Florida, 2025”

They also explore the growing role of parents as advocates and innovators, the case for a national rare disease champion, and why a more joined-up approach across government, regulators, industry and charities is essential to delivering faster diagnoses and access to life-changing therapies. To mark Rare Disease Day, this special Spotlight episode explores why rare diseases – affecting an estimated one in 17 people in the UK – still face years of diagnostic delays and limited treatment options. Why does diagnosis take so long?

Original title: “Rare diseases: from lived experience to lasting treatments - New Statesman”

They also explore the growing role of parents as advocates and innovators, the case for a national rare disease champion, and why a more joined-up approach across government, regulators, industry and charities is essential to delivering faster diagnoses and access to life-changing therapies. To mark Rare Disease Day, this special Spotlight episode explores why rare diseases – affecting an estimated one in 17 people in the UK – still face years of diagnostic delays and limited treatment options. Why does diagnosis take so long?

Original title: “Rare diseases: from lived experience to lasting treatments - New Statesman”

Over half of biopharma professionals would work again at the companies that let them go, according to a BioSpace LinkedIn poll. Several professionals, as well as recruiting and talent acquisition experts, discuss reasons for—and a key risk of—going back.

Original title: “Keeping the Door Open: Many Would Return to Companies That Laid Them Off”

“It was the OI Foundation that really helped my family navigate my diagnosis, and I grew up going to the national conferences,” said Fynan. “It was becoming a mom of daughters with OI that really lit a fire underneath me to see how advocacy can be more than just another branch of what we are doing. It can be that acceleration for moving in the right direction in rare disease... “It was the OI Foundation that really helped my family navigate my diagnosis, and I grew up going to the national conferences,” said Fynan. “It was becoming a mom of daughters with OI that really lit a fire underneath me to see how advocacy can be more than just another branch of what we are doing. It can be that acceleration for moving in the right direction in rare disease.” “We also advocate for rare disease education within our education, ensuring that Quinnipiac students enter the workforce prepared to help patients with rare diseases.” · Students for Rare was supported in hosting this event by their faculty adviser, Maureen Helgren, who is an associate professor of medical sciences. Helgren expressed her fulfillment in advising the Students for Rare group. “The reality is that it is hard to live with a rare disease and to care for family and friends with a rare disease. Organizations such as NORD or disease-specific foundations began and continue to support the rare disease community,” said Helgren. Quinnipiac Students for Rare, a chapter of the National Organization for Rare Disorders (NORD), hosted its annual Rare Disease Day on the North Haven Campus on February 20 focusing on a condition called Osteogenesis Imperfecta (OI). With panelists ranging from research experts and clinicians to patients living with rare diseases, the event enriched students’ understanding of rare diseases and how they can support patients with rare diseases as future healthcare professionals. The center is nationally recognized and hosts many events throughout the year, with many focused on rare disease. The Center for Interprofessional Healthcare Education has encouraged students from various programs to collaborate as they prepare to support their patients.

Original title: “Student-led Rare Disease Day calls community to action | Quinnipiac Today”

Describes mosquito-borne diseases and how to protect workers.

Original title: “Mosquito-Borne Diseases in Workers”

An emotional plea from a family in Ottawa fighting to save their four year old daughter with a rare condition that will rob her of all her basic skills and abilities. The girl's parents say a clinical... An emotional plea from a family in Ottawa fighting to save their four year old daughter with a rare condition that will rob her of all her basic skills and abilities. The girl's parents say a clinical trial could save her life, but they recently learned she isn't eligible for it.

Original title: “Family fights for gene therapy for child with rare disease - Yahoo News Canada”

This webinar explores how collaborative-initiated trials and Investigator-Initiated Trials (IITs) are complementing traditional drug development to accelerate innovation, advance precision medicine, and address clinically meaningful questions.

Original title: “Webinar: The Future is Collaborative: Transforming Clinical Trials”