Former ACIP vice chair Robert Malone claimed that Andrew Nixon, spokesperson for the Department of Health and Human Services, “trashed” him with the media, adding that he resigned because “I do not like drama.”

Original title: “After Speaking out of Turn, Malone Steps Down From Vaccine Committee”

CHI Development Director Pam Weber-Leaf shares her experience at the 2026 Rare Disease Day event sponsored by NORD. CHI Associate Board Co-Chairs Amy Patterson and Hanna Raskin-Gross (L-R in first photo) at Johns Hopkins University Hospital’s Rare Disease Day event. That same day – Friday, Feb. 27th — CHI Associate Board Co-Chairs Amy Patterson and Hannah Raskin-Gross celebrated RDD at Johns Hopkins University Hospital! Amy is a genetics counselor at Johns Hopkins and led the event, which featured a full day of speakers and an advocacy fair for patient advocacy groups. CHI had a chance to raise hyperinsulinism (HI) awareness and connect with our peers in the rare disease space all over the Northeast corridor of the United States last month as part of international Rare Disease Day (RDD) 2026 observances. After welcome remarks by center Co-Director and genetics Professor Yong-hui Jiang about innovations in genetic testing, I was pleased to hear NORD’s Darby Gavin speak about the importance of collaboration between rare disease organizations to track data that drives broad-based policy change related to diagnosis and treatment access. Thunderstorms that evening clearly hadn’t deterred families from making the trek to New Haven from all over the area, and when the kids weren’t doing organized activities, they had a blast running around among the life-sized dinosaur models in the adjacent exhibition hall. Animals of another stripe – and not the prehistoric kind — were also plentiful: stuffed zebras, the official mascot of rare diseases, were showing their stripes on every table.

Original title: “2026 Rare Disease Day Roundup - Congenital Hyperinsulinism International”

For years, I knew something wasn’t right. I was experiencing symptoms that affected my daily life—shortness of breath, fatigue, and physical limitations that didn’t make… Continue reading The Diagnosis They Missed: A Journey from Dismissal to Advocacy I speak about the gaps that exist between patients and healthcare systems. I share the emotional and mental toll of living with chronic illness—the part that often goes unseen. And I work to help healthcare providers and organizations better understand what it truly means to live with a rare condition. My hope is that by sharing my story, others will feel less alone—and that those in positions to make change will listen more closely, act more compassionately, and recognize the value of the patient voice. No one should have to fight to be believed. Podcast: I’m Aware That I’m Rare: the phaware® podcast It took years before I finally received answers. By the time I was diagnosed with pulmonary hypertension—a rare and serious condition—my reality had already changed. What I had been feeling all along was real. The signs were there. Through this journey, I realized that so many people living with rare and chronic conditions face the same experience: delayed diagnosis, being unheard, and navigating a healthcare system that isn’t always equipped to recognize complex illnesses.

Original title: “The Diagnosis They Missed: A Journey from Dismissal to Advocacy - Rare Disease Day 2026”

Among biopharma professionals surveyed by BioSpace, respect remains higher for managers than for CEOs, and unhappiness with company leadership had nearly 1 in 7 people looking to walk away from their employer.

Original title: “Leadership, Not Managers, More Likely To Drive Employees Away, BioSpace Finds”

/PRNewswire/ -- The National Organization for Rare Disorders (NORD®) today announced the appointments of Michael J. Beard as Vice President of Federal and... WASHINGTON, March 25, 2026 /PRNewswire/ -- The National Organization for Rare Disorders (NORD®) today announced the appointments of Michael J. Beard as Vice President of Federal and Global Public Policy and Cara Tenenbaum, JD, MBA, as Director of Regulatory Affairs, further strengthening the organization's leadership and long-standing commitment to advancing policy on behalf of the rare disease community. ... NORD® welcomes Michael J. Beard as Vice President of Federal and Global Public Policy and Cara Tenenbaum JD, MBA, as Director of Regulatory Affairs, further strengthening the organization's leadership in advancing patient-centered policy for the more than 30 million Americans living with rare diseases, over half of whom are children. NORD® welcomes Michael J. Beard as Vice President of Federal and Global Public Policy and Cara Tenenbaum JD, MBA, as Director of Regulatory Affairs, further strengthening the organization's leadership in advancing patient-centered policy for the more than 30 million Americans living with rare diseases, over half of whom are children. These appointments build on NORD's sustained investment in policy leadership and reflect a continued focus on advancing coordinated, high-impact strategies across legislative and regulatory priorities, both for individual patients and families and for the hundreds of patient advocacy organizations that rely on NORD as a trusted policy partner. Cara Tenenbaum, JD, MBA, joins NORD as Director of Regulatory Affairs, bringing nearly two decades of experience at the intersection of federal policy, regulatory strategy, and patient advocacy. A former leader at the FDA and a trusted advisor to patient organizations and health innovators, she will lead NORD's regulatory policy efforts, advancing strategies that support the development, approval, and access to safe and effective treatments for people living with rare diseases. About NORD® The National Organization for Rare Disorders (NORD®) is the leading independent nonprofit organization representing the more than 30 million Americans with rare diseases and supporting hundreds of patient advocacy organizations nationwide.

Original title: “NORD Advances Policy Leadership with Strategic Appointments to Strengthen Rare Disease Advocacy”

WASHINGTON, D.C. — March 24, 2026 — The National Organization for Rare Disorders (NORD®) today announced the appointments of Michael J. Beard as Vice President of Federal and Global Public … Continue reading "NORD Advances Policy Leadership with Strategic Appointments to Strengthen Rare Disease Advocacy"

Original title: “NORD Advances Policy Leadership with Strategic Appointments to Strengthen Rare Disease Advocacy”

On a crisp March morning in New York City, something powerful unfolded, not just on the racecourse, but within a community united by purpose.  The EveryLife Foundation for Rare Diseases […] The post Running for Something Greater: EveryLife Foundation’s Inaugural Team at the United Airlines NYC Half Marathon appeared first on EveryLife Foundation for Rare Diseases.

Original title: “Running for Something Greater: EveryLife Foundation’s Inaugural Team at the United Airlines NYC Half Marathon”

Bartonellosis is a disease caused by <em>Bartonella </em>bacteria spread by fleas, body lice, and sand flies.

Original title: “About <em>Bartonella</em>”

Sepsis is a medical emergency. Learn the risks, spot the signs and symptoms, and act fast.

Original title: “About Sepsis”

Ways parents and caregivers can help children have a healthy weight and establish healthy habits.

Original title: “Preventing Childhood Obesity: 6 Things Families Can Do”

Learn more about the diagnostic odyssey and how Europe can lead the path to its improvement The post Ending the diagnostic odyssey: How Europe can lead the way in rare disease diagnosis appeared first on EURORDIS-Rare Diseases Europe.

Original title: “Ending the diagnostic odyssey: How Europe can lead the way in rare disease diagnosis”

Recruiters can play a significant role in biopharma professionals getting hired, especially in an employer-driven job market. However, when working with them, candidates need to avoid making six key mistakes, from waiting too long to ask for help to prematurely contacting hiring companies.

Original title: “6 Things Candidates Do That Make It Harder for Recruiters To Get Them Jobs”

Their child has lived with the rare disease since birth, and now they're turning that experience into a message for other families. WALB's Brittanye Blake has their story. Through social media content, the family has been able to educate healthcare professionals on the rare disease. Smiechowski says she’s received comments from health professionals and, more specifically, NICU nurses who have seen similar cases like her sons. “It just warms my heart because we are making a difference in some way,” she said. Now, the family is bringing their online mission to real life by launching a nonprofit called Beyond the Cry Foundation. A mission that started as “Mornings with the Smiechowski’s” on TikTok has grown to a platform that raises awareness about the disease and makes connections with other families facing the same challenges. Sara Smiechowski and her husband had a change when their third child, Mathias, was born with a rare genetic condition called Cri du Chat syndrome, also known as Cat’s Cry.(Sara Smiechowski) Their child has lived with the rare disease since birth, and now they’re turning that experience into a message for other families. The nonprofit foundation is set to launch on April 3.

Original title: “‘We are making a difference in some way’: South Georgia family launches foundation to raise awareness about their son’s rare disease”

Applications for the #RAREis Scholarship Fund are now open. Established in 2020, the #RAREis Scholarship Fund supports individuals living with rare diseases as they pursue their educational goals. This year, […] The post #RAREis Scholarship Fund Surpasses 500 Awards as 2026 Applications Open appeared first on EveryLife Foundation for Rare Diseases.

Original title: “#RAREis Scholarship Fund Surpasses 500 Awards as 2026 Applications Open”

Honest and open conversations, both with your provider and sexual partner(s), are important

Original title: “Conversation Tips”

Discover what PE fellows learn and do as part of the fellowship.

Original title: “What Fellows Do”

Hydrocephalus. Ataxia. Dandy-Walker syndrome. Agenesis of the corpus callosum. Hypotonia. Macrocephaly. Cortical visual impairment. Global developmental delay. Failure to thrive. Unilateral Renal hypoplasia. Congenital inguinal… Continue reading Acceptance without Answers We were referred to the undiagnosed diseases network when he was a toddler. This time, we would finally figure out why our son was facing so many challenges, right? Nope; not yet at least.

Original title: “Acceptance without Answers - Rare Disease Day 2026”

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

Original title: “Infants, Toddlers, and Children”

As the Formula 1 season kicks off in the APAC region, biopharma companies can take a page from the popular sport’s playbook around precision, adaptability, and the right team. In return, biopharma can also offer some lessons to elite motor sport on well-rounded excellence in the race to market.

Original title: “What Biopharma Can Learn from Formula 1: Precision, Speed, and the Power of the Right Partner”