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,…
Sanofi has announced encouraging results from a Phase 2 clinical trial of efdoralprin alfa, an investigational therapy for alpha-1 antitrypsin deficiency (AATD), a rare genetic disorder affecting the liver and…
Sanofi has announced encouraging results from its Phase 2 ElevAATe clinical trial evaluating efdoralprin alfa (SAR447537), a recombinant human alpha-1 antitrypsin (AAT)-Fc fusion protein, for the treatment of emphysema caused…
Genentech, a member of the Roche Group, has unveiled results from two pivotal Phase III trials—MEERKAT and SANDCAT—evaluating the investigational drug vamikibart for uveitic macular edema (UME), a sight-threatening complication…
According to a press release from Syndax Pharmaceuticals, the developer of Revumenib, the drug met its primary endpoint resulting in complete remission (CR) or CR with partial recovery. Relapsed or…
Initial results from the TROPION-PanTumor03 phase 2 trial, presented at the 2025 ESMO Congress, show that the combination of DATROWAY® (datopotamab deruxtecan) and rilvegostomig offers significant tumor responses and disease…
Astellas Pharma Inc. has announced that its Phase 2 GLEAM trial evaluating zolbetuximab in combination with gemcitabine plus nab-paclitaxel for metastatic pancreatic adenocarcinoma did not meet its primary endpoint of…
AstraZeneca and Daiichi Sankyo have announced promising Phase 3 trial results for Datroway, their investigational antibody-drug conjugate (ADC) targeting triple-negative breast cancer (TNBC). The trial demonstrated that Datroway significantly slowed…
Bayer AG and its subsidiary BlueRock Therapeutics LP have announced encouraging 36-month follow-up data from the Phase I exPDite clinical trial of bemdaneprocel (BRT-DA01), an investigational cell therapy aimed at…
Capsida Biotherapeutics has suspended a clinical trial of its experimental gene therapy after the first child to receive the treatment died, sending shockwaves through the rare disease and gene therapy…
Serina Therapeutics, a clinical-stage biotechnology company, has announced a major development in the treatment of Tardive Dyskinesia (TD) by advancing its innovative POZ-VMAT2i candidate into clinical development. This move, reported…
Cogent Biosciences has announced new clinical trial results indicating that its drug BEZUCLASTINIB offers promising benefits for patients with indolent systemic mastocytosis (ISM), a rare and chronic disorder characterized by…
Do you know about Lafora Disease? This rare genetic disease begins during adolescence. Your child starts their life healthy and whole, but then they decline, cognitively and physically, until they…
The results released from the Phase III ESSENCE clinical trial, and recently published by Medical Xpress and in the NEJM, found that the drug semaglutide is effective in the treatment…
Humans have been using tobacco as a drug for more than 12,000 years. It wasn't until the early-20th century that the link between tobacco and lung cancer (and other health problems)…
Acknowledgment: This story is sponsored by Alexion Pharmaceuticals and is promoted through the Patient Worthy Collaborative Content program. We only publish content that embodies our mission of providing relevant, vetted,…
Approximately one and one half million people are affected by ulcerative colitis (UC) worldwide. It is an inflammatory bowel disorder with, as of yet, no known cause. There are, however,…
Imagine that your loved one is diagnosed with a rare disease. The first thing you do is to go to the internet to understand what you can about this disease…
By: Harsha K Rajasimha, Ph.D., Founder and CEO of Jeeva Clinical Trials Challenges Unique to Cell and Gene Therapies (CGTx) Clinical Trials Cell and gene therapies (CGTx) have emerged as…
Rare disease drug development can be challenging. Countries globally developed programs to incentivize innovation and development in this space. One of these initiatives is the designation of orphan drugs, or…
Clinical-stage biopharmaceutical company Savara Inc. was founded on a desire to develop innovative therapies for people living with rare respiratory diseases. The company's work over the past few years centers…
Ocugen, Inc., a biotechnology company based in Malvern, Pa. recently reported to BioSpace that DSMB gave a favorable review of its Phase ½ ArMaDa clinical study of OCU410. Approximately 10…
Long COVID is a phenomenon emerging from the global spread of SARS-CoV-2, a strain of coronavirus causing COVID-19. Seven million people died from COVID-19, though this is considered a serious…
Mirum Pharmaceuticals has long been a leader in the rare liver disease space, driving forward with a mission of empowering patients and families, and identifying novel therapeutic interventions to help…
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