Researchers at the University of Southampton have uncovered a hidden culprit in blood cancer, a unique sugar molecule that disguises aggressive lymphoma cells and renders them resistant to conventional treatments.…
A new artificial intelligence model, popEVE, could accelerate diagnoses for rare, single-variant genetic diseases by scoring how likely each genetic change in a patient’s genome is to cause disease. Developed…
University of Southampton researchers have identified a distinct subtype of diffuse large B‑cell lymphoma (DLBCL), termed “Mann-type DLBCL,” marked by a unique mannose sugar on the B‑cell receptor that fuels…
Three-year-old Oliver Chu has stunned clinicians after becoming the first person with Hunter syndrome (MPS II) to receive an experimental gene therapy and showing striking early gains. Hunter syndrome is…
Hundreds more patients in England with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) will gain earlier access to glofitamab (Columvi) after new guidance from the National Institute for Health…
Vera Therapeutics has taken a major step toward transforming treatment for immunoglobulin A nephropathy (IgAN), submitting a Biologics License Application to the U.S. Food and Drug Administration through the Accelerated…
Arrowhead Pharmaceuticals has secured its first U.S. approval with Redemplo, an RNA interference therapy cleared by the FDA for adults with familial chylomicronemia syndrome (FCS). Used alongside dietary management, as…
See additional Important Safety Information below. Please read the accompanying full Prescribing Information, including Boxed WARNING, also available at NUPLAZID.com. You are encouraged to report negative side effects of prescription…
Maple syrup urine disease (MSUD) represents one of medicine's most deceptive challenges, a rare inherited metabolic disorder where patients can appear dangerously well while their brains swell dangerously. As reported…
The NHS has dramatically expanded access to a life-saving cancer therapy, according to NHS England, offering approximately 300 blood cancer patients in England annually a genuine chance at complete remission.…
A groundbreaking achievement in genetic medicine has emerged as the European Medicines Agency (EMA) recommended approval of Waskyra (etuvetidigene autotemcel), the first gene therapy specifically designed to treat Wiskott-Aldrich syndrome…
Hypereosinophilic syndrome (HES) patients may finally have a meaningful treatment option. According to an article published at BusinessWire.com, AstraZeneca's FASENRA (benralizumab) has shown statistically significant efficacy in delaying disease progression,…
Tonix Pharmaceuticals announced plans to initiate Phase 2 clinical trials in 2026 for TNX-2900, an innovative intranasal oxytocin formulation designed to treat Prader-Willi Syndrome (PWS), a rare genetic disorder characterized…
Takeda Pharmaceutical Company announced promising interim data from a Phase 1b clinical trial demonstrating that mezagitamab (TAK-079), an experimental immunotherapy, maintains kidney function stability for extended periods even after treatment…
Biohaven Pharmaceuticals faced a significant setback when the U.S. Food and Drug Administration issued a Complete Response Letter (CRL) rejecting the company's New Drug Application for Vyglxia (troriluzole), a promising…
The U.S. Food and Drug Administration has approved UCB's Kygevvi, marking a historic milestone for patients with thymidine kinase 2 deficiency (TK2d), an ultra-rare inherited mitochondrial disorder that has lacked…
Editor’s Note: This story is a continuation of Jenny Decker’s global sailing voyage. To see the original article, please click here. Jenny Decker's journey called “Just a Lap" continues. Patient…
There's a lot to consider when you’re facing tumor removal surgery. Think about mandible or jaw surgery, for example. Your face is part of every interaction you have, how you…
Soleno Therapeutics marked a significant turning point in its corporate history by reporting its first profitable quarter, driven by robust sales of VYKAT XR, its groundbreaking therapy for Prader-Willi Syndrome…
A once-rare condition that causes severe allergic reactions to red meat, known as alpha-gal syndrome (AGS), is experiencing a dramatic rise across the United States, according to new research from…
vTransthyretin amyloid cardiomyopathy (ATTR-CM) is a progressive heart condition driven by the buildup of abnormal transthyretin protein in the heart muscle and, at times, in nerves. For many living with…
A groundbreaking discovery by an international team led by Shinghua Ding at the University of Missouri has revealed a previously unknown genetic disorder affecting motor neurons and muscle control. As…
BridgeBio Pharma has marked another significant milestone in rare disease research, announcing a second late-stage clinical trial success—this time with encaleret, a therapy for the uncommon endocrine disorder autosomal dominant…
At the 67th American Society of Hematology (ASH) Annual Meeting, Gilead Sciences and its subsidiary Kite showcased a suite of new data underscoring major progress in cell therapy for blood…
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