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…
A gene-editing protein, small enough to fit into adeno-associated viruses (AAVs) that infect humans has been identified. As reported by Precision Medicine, these compact AAVs can be modified so that…
I was introduced to Amber Olsen, the “mama bear” in my new documentary film, The Zebra and the Bear, in the spring of 2016. A mutual friend asked if I…
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…
As reported on PharmaBiz, the National Organization for Rare Disorders (NORD) has announced a new request for proposals (RFP) aimed at nonprofit patient advocacy groups interested in launching rare disease…
The developer of the Promega OncoMate MSI Dx Analysis System recently announced the FDA’s approval of the system identifying patients who may benefit from the combination of Lenvatinib (Lenvima) and…
As reported on World Pharma News, a collaborative research team from the Medical University of Vienna, HUN-REN Research Centre for Natural Sciences, and Eötvös Loránd University has unveiled a promising…
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…
At the 76th annual AASLD Liver Meeting®, Novo Nordisk presented new findings from a post hoc analysis of the ESSENCE phase 3 trial, highlighting the impact of semaglutide 2.4 mg…
Nexiguran ziclumeran (nex-z), an investigational CRISPR-Cas9 gene therapy for transthyretin amyloid cardiomyopathy (ATTR-CM), continues to demonstrate long-term efficacy in early-phase research, even as its phase III trials remain on hold…
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…
Why Rare Diseases Pose a Diagnostic Challenge As discussed in the Orphanet Journal of Rare Disease, a "rare disease" is defined as a condition affecting fewer than 1 in 2,000…
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…
Please go HERE to read Part 1 of this article The company, GEMMABIO, will pursue a number of rare, large indications. A new spinout, RareTx, will be dedicated to Orphan…
My name is Danielle, and in 2021, I was diagnosed with Stage 3 MASH, a life-threatening liver disease. I consider myself lucky because I had no real symptoms; my MASH was…
Written by Diane Wilkie My hATTR story began in the mid-1960’s when my father, a healthy, strong, outdoorsy guy in his late 40’s started tripping, experiencing numbness, and having dizzy…
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…
Prader-Willi syndrome (PWS) is a rare genetic disorder affecting approximately 1 in 10,000–30,000 individuals worldwide. It is the leading syndromic cause of obesity, with an estimated 400,000 cases globally and…
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…
By Thresa Giles, CEO — Hope & Help Center of Central Florida Since HIV was first identified in 1983, there has been a growing, persistent stigma surrounding it. People have…
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…
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