What's the Good News? Researchers have successfully completed initial testing of an experimental Parkinson's medication called TT-P34. The early results are very encouraging and suggest the drug is safe, works…
The history of cystinosis is one of the most successful examples of rare disease research leading to meaningful treatments. In a recent interview with the European Medical Journal, William Gahl—a…
This is my son Nikolai. Nikolai was recently diagnosed with Mucopolysaccharidosis type 2, or "Hunter Syndrome." A rare genetic form of childhood dementia, it's caused by a mutation in the…
As reported on PharmaBiz, a combination of Imfinzi (durvalumab) and Imdelltra (tarlatamab) has delivered significant improvements in survival outcomes for patients with extensive-stage small cell lung cancer (ES-SCLC), according to…
At the age of 18, I was diagnosed with a rare swallowing disorder called Achalasia. At the time of this video, I had undergone several surgeries, procedures, and countless appointments,…
In honor of World Duchenne Awareness Day on September 7th, Mal Duffin has invited us to share this app designed for parents whose children are living with Duchenne Muscular Dystrophy. Mal's…
As reported on World Pharma News, the U.S. Food and Drug Administration has granted approval to daraxonrasib, an oral RAS-targeting therapy, for adults with metastatic pancreatic adenocarcinoma who have previously…
A novel investigational Ebola vaccine has shown encouraging safety and immunogenicity results in a first-in-human clinical trial, supporting further development of an alternative vaccination strategy against Ebola virus disease, as…
Editor's Note: Patient Worthy is honored to share this article, originally written by David St. Denis, President & Director at Anteris Technologies. Transcatheter aortic valve replacement (TAVR) was originally developed…
Although Nancy Wexler’s efforts were largely responsible for creating a genetic test to locate the Huntington’s gene, she refused to take the test herself saying “I will meet the devil…
As reported on Fierce BioTech, Guardant Health has secured U.S. Food and Drug Administration approval for its Guardant360 CDx liquid biopsy test as the companion diagnostic for AstraZeneca’s newly approved…
As reported on Science Daily, researchers at the University of Michigan have identified a potential new therapeutic approach for aggressive prostate cancers that become resistant to standard hormone-targeting treatments. Preclinical…
GPRC5D-Targeting Approach Positions Company to Expand Post-BCMA Treatment Options As reported on Fierce BioTech, Bristol Myers Squibb announced positive results from its pivotal Quintessential trial evaluating arlocabtagene autoleucel, a genetically…
Editor's Note: We're honored to share the following article from our friends at the Courageous Parents Network. To see this article in its original format, please click here. Parents will…
Editor's Note: Patient Worthy is honored to share this article from Rareatives, originally written by our friend Jessica Lynn. To see the article in its original format, please click here.…
Editor's Note: Patient Worthy is honored to share this story from our friends at Elephants & Tea, originally written by Bill Phillips, a colorectal cancer survivor. To see the article…
A single genetic change can have dramatically different effects from one person to the next. That is the reality for people living with PTEN hamartoma tumour syndrome (PHTS), a rare…
As reported on QZ, the U.S. Food and Drug Administration has approved a new indication for Eli Lilly’s tirzepatide (Mounjaro), allowing the therapy to be used to reduce the risk…
As reported on MedCity News, the U.S. Food and Drug Administration has approved Takeda Pharmaceutical’s Mimrylo (rusfertide) for the treatment of erythrocytosis in adults with polycythemia vera (PV), a rare…
As reported on Drugs, BioNTech has decided to halt development of its Phase 2 BNT122-01 study investigating autogene cevumeran (BNT122/RO7198457) in patients with resected colorectal cancer who remained ctDNA-positive after…
As reported on PharmaBiz, BeOne Medicines has reported positive results from the second interim analysis of the phase 3 HERIZON-GEA-01 trial, further supporting the use of zanidatamab (Ziihera)-containing regimens as…
At 22, a young person's world should be filled with possibilities—a career to build, places to travel, friendships to make, and a future to imagine. Instead, this young man, David,…
As reported on Pharmaceutical Technology, the US Food and Drug Administration (FDA) has granted approval to PharmaEssentia’s Besremi (ropeginterferon alfa-2b-njft) for the treatment of adults with essential thrombocythaemia (ET), providing…
In a recent press release, Novartis has reported positive topline results from its Phase III REMODEL-1 and REMODEL-2 trials, showing that the investigational oral Bruton’s tyrosine kinase (BTK) inhibitor remibrutinib…
As reported on Business Wire, the European Commission has approved trastuzumab deruxtecan (Enhertu) in combination with pertuzumab for the first-line treatment of adults with unresectable or metastatic HER2-positive breast cancer.…
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