As reported on BioPharmaDive, new clinical trial data presented at the American Society of Hematology (ASH) annual meeting suggests that an early treatment regimen combining Tecvayli (Johnson & Johnson’s bispecific…
Key Insight: As reported by Healio, metabolic dysfunction-associated steatotic liver disease (MASLD) is not exclusive to individuals with obesity. New research presented at The Liver Meeting reveals that lean patients…
As reported on MedicalXpress, esearchers at the Children’s Hospital of Philadelphia (CHOP) and the University of Pennsylvania are pioneering a new approach to treating rare genetic disorders using CRISPR-based therapies.…
Chiesi Group has entered an exclusive licensing agreement with Aliada Therapeutics, a subsidiary of AbbVie, to develop therapies that can cross the blood-brain barrier (BBB) for lysosomal storage disorders (LSDs).…
As reported on Healio, new findings presented at The Liver Meeting highlight the potential of resmetirom (Rezdiffra, Madrigal Pharmaceuticals) in managing metabolic dysfunction-associated steatohepatitis (MASH) with compensated cirrhosis—a population with…
As reported on NPR, Tatiana Schlossberg, journalist and granddaughter of former U.S. President John F. Kennedy, has disclosed that she is battling a rare and aggressive form of leukemia. In…
As reported on BioSpace, the U.S. Food and Drug Administration (FDA) has launched an investigation into reports of serious adverse events, including one fatality, associated with Takeda’s enzyme replacement therapy…
The United Leukodystrophy Foundation (ULF) is proud and excited to announce a partnership with Marc Nolan, a Chicago-based footwear brand, who is launching a new campaign with us “Celebrating life’s…
The U.S. Food and Drug Administration (FDA) has approved Koselugo (selumetinib), an oral MEK inhibitor developed by Alexion, AstraZeneca Rare Disease, for adults with neurofibromatosis type 1 (NF1) who have…
My journey started abruptly last year. It was a completely normal day—until it wasn't. I'm an RN, and I slept most of the day before my night shift. I talked…
As reported by PharmaBiz, the European Medicines Agency’s Committee for Orphan Medicinal Products (COMP) has issued a positive opinion supporting Orphan Drug Designation (ODD) for BioInvent’s BI-1808, an investigational anti-TNFR2…
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…
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…
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…
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…
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…
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…
As reported on MSN, a preliminary study presented at the American Heart Association’s Scientific Sessions 2025 suggests that a non-invasive method for assessing atrial function could help identify stroke risk…
Merck & Co. announced positive results from two Phase 3 studies of its kidney cancer drug Welireg, signaling potential expansion into earlier stages of disease. As reported on BioPharmaDive, in…
A Wake Forest University School of Medicine study reports that two existing medications (empagliflozin (Jardiance), a diabetes drug, and intranasal insulin) could improve brain health in individuals with mild cognitive…
We're honored to bring you this video, shared with us by Danny Gereg, that details some of his journey living with bladder cancer. Danny has stayed resilient and positive, finding…
Research into amyotrophic lateral sclerosis (ALS) is gaining momentum. According to Citeline, early-stage clinical activity surged from 28 new trials in 2023 to 60 in 2024, a remarkable increase for…
Cowden Syndrome. A name that sounds almost gentle, until you realize it carries generations of uncertainty. It’s caused by changes in the PTEN gene and can affect the skin, thyroid,…
As reported by drugs.com, at the American College of Rheumatology (ACR) Convergence congress, Novartis presented encouraging results from its Phase III NEPTUNUS-1 and NEPTUNUS-2 trials, highlighting the potential of ianalumab…
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