According to a story from BioPortfolio, the drug development company Dicerna Pharmaceuticals recently announced the release of proof of concept data in its Phase I trial testing the company's experimental…
A phase 1/2 study of the drug lumasiran for the treatment of primary hyperoxaluria Type 1 has produced encouraging results, reports Alnylam Pharmaceuticals. The full article can be read here,…
Dicerna Pharmaceuticals has dosed the first patient with primary hyperoxaluria in Group B of a clinical trial of the investigational drug DCR-PHXC. Dicerna has also announced that DCR-PHXC has just…
This past July, professionals, patients, and families with loved ones who struggle with primary hyperoxaluria Type 1, 2 and 3 were offered a glimpse of hope and a better future.…
Primary Hyperoxaluria Type 1 (PH1)What is primary hyperoxaluria type 1 (PH1)?Primary hyperoxaluria type 1 (PH1) is a rare disorder that affects the kidneys. PH1 results from buildup of oxalate, which normally…
Last week, from October 22nd through the 25th, marked the American Society of Nephrology (ASN) Kidney Week. According to a press release, Synthetic Biotic medicine developer Synlogic, Inc. ("Synlogic") presented…
Alnylam Pharmaceuticals has just announced that they will be starting a Phase 2 trial for their RNAi therapeutic called Lumasiran. This trial will investigate both the safety as well as…
February 28th is known as Rare Disease Day, designed to raise awareness of rare diseases, patients, caregivers, and their experiences. As this day approaches, the American Kidney Fund (AKF) shared…
Sign Up With a Patient Worthy Account and Share Your Rare Story
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
