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…
Aldeyra Therapeutics, Inc., a biotechnology leader in immune-mediated disease therapies, has announced encouraging results from its Phase 2 clinical trial evaluating ADX-629 in patients with alcohol-associated hepatitis, and is now…
Halia Therapeutics, Inc., a clinical-stage biopharmaceutical company based in Utah, has announced that its investigational drug, ofirnoflast (HT-6184), has received Orphan Drug Designation (ODD) from the U.S. Food and Drug…
Older adults are at heightened risk for severe anaphylaxis but are less likely than younger adults to receive the recommended treatment of epinephrine, according to findings from two recent studies…
Grieving families who have lost loved ones to severe allergic reactions are calling for urgent reforms to the way deaths from anaphylaxis are investigated in England and Wales. These families,…
BridgeBio Pharma has reported decisive success in a pivotal phase 3 trial for its rare disease drug BBP-418, aimed at treating limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9). Reported by Fierce…
Merck & Co has taken a significant step forward in its immunology pipeline by initiating Phase IIb clinical studies for tulisokibart (MK-7240), its investigational therapy targeting immune-mediated inflammatory diseases. According…
This patient story is sponsored by AVEO Pharmaceuticals, Inc. and is promoted through the Patient Worthy Collaborative Content program. We only publish content that embodies our mission of providing relevant, vetted,…
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