Your story has the power to educate, motivate, and make a real difference in the lives of others. We are looking for patients with Metabolic Dysfunction-Associated Steatohepatitis, or MASH –…
In a groundbreaking international effort led by Dr. Shady Rahayel of Université de Montréal and reported by MSN.com, researchers have discovered new ways to predict which individuals with isolated REM…
Clinical trials often begin with optimism, sites are launched, projections look promising, and timelines are set. But when enrollment stalls, sponsors frequently call-in patient recruitment partners as a last-minute “rescue”…
Doctors and researchers at the University of Colorado Anschutz Medical Campus have achieved a groundbreaking milestone by employing advanced Neuropixels technology in the study of Parkinson’s disease. Reported by Pharmabiz.com,…
In a significant advancement for rare disease treatment in Japan, the Ministry of Health, Labour and Welfare (MHLW) has approved Bylvay® (odevixibat) for the management of pruritus associated with progressive…
In a landmark step for personalized cancer care, the National Comprehensive Cancer Network® (NCCN®) has launched the NCCN Guidelines Navigator™—a cutting-edge digital platform that redefines how clinicians access and use…
Your story has the power to educate, motivate, and make a real difference in the lives of others. We are looking for patients with Metabolic Dysfunction-Associated Steatohepatitis, or MASH -…
A new analysis has uncovered stark differences in the rates of breast anaplastic large cell lymphoma (ALCL), a rare cancer often linked to breast implants, among women of different racial…
A recent preprint reported by medRxiv explores the complex relationship between genetic mutations and clinical presentation in Colombian patients with congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency (21OHD). While…
Alera Bio, a Chicago-based biotech company dedicated to rare neurological diseases, has announced a significant breakthrough in the quest to treat MCT-8 deficiency, otherwise known as Allan-Herndon-Dudley Syndrome. On September…
When Ashley Rowland’s daughter, Aubrie, was diagnosed with a rare mitochondrial disease called AARS2-related leukoencephalopathy before her first birthday, Ashley’s world changed forever. Seven years later, she continues her fight—not…
Rare cancers, defined by their low incidence, collectively constitute nearly a quarter of all cancer diagnoses in the US and Europe, and represent a significant public health challenge. Despite their…
A major new synthesis of research has confirmed that people living with antineutrophil cytoplasmic antibody (ANCA)-associated vasculitis (AAV) face a markedly increased risk of developing cancer. Published in Zeitschrift für…
Recent research has brought to light striking genetic differences in chronic lymphocytic leukemia (CLL) among African American (AA) patients, suggesting that their disease may follow a distinct course compared to…
Misfolded proteins found in motor neurons are the cause of muscle weakness and paralysis. People with Amyotrophic Lateral Sclerosis (ALS) generally live two to four years after the onset of…
A recent study published in Clinical Lymphoma, Myeloma, and Leukemia offers new hope for older adults with myelofibrosis (MF), suggesting that allogeneic hematopoietic stem cell transplantation (allo-HCT) remains a viable,…
JCR Pharmaceuticals Co., Ltd., a leader in therapies for rare and genetic diseases, has reached an important milestone in its ongoing collaboration with Alexion, AstraZeneca’s rare disease division. The achievement,…
Jazz Pharmaceuticals plc has taken a major leap in rare cancer treatment with the recent FDA accelerated approval of Modeyso (dordaviprone), the first-ever therapy for relapsed H3 K27M-mutated diffuse midline…
On September 3, 2025, the US Food and Drug Administration (FDA) introduced the Rare Disease Evidence Principles (RDEP) process, a groundbreaking step for drug development targeting ultra-rare genetic diseases. According…
A groundbreaking study from the Molecular Oncology Research Center at Hospital de Amor in Barretos, Brazil, is paving the way for more personalized and less toxic treatments for children with…
Hemolytic disease of the fetus and newborn (HDFN) stands as one of perinatal medicine’s most complex challenges. A recent systematic review, published in the Journal of Perinatology by Verweij, Lopriore,…
Learning that your child has neurofibromatosis type 1 (NF1) can be overwhelming. NF1 affects each child differently and brings uncertainty, but with the right knowledge, resources, and support, parents can…
MyOme, a Menlo Park leader in clinical whole genome analysis, has announced a significant upgrade to its rare disease testing platform, now offering tandem repeat expansion (TRE) and mitochondrial genome…
Luke McCarthy of Killybegs is preparing to take on the Galway Bay Marathon, not just as a personal fitness goal, but as a heartfelt tribute to his father, Joe, who…
On Sunday, more than 100 medical experts from across the globe gathered in Rochester for the Mayo Clinic’s four-day “Undiagnosed Hackathon”—a pioneering event dedicated to solving rare diseases that have…
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