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…
In late November, RNAi therapeutics company Alnylam Pharmaceuticals ("Alnylam") announced that its therapy OXLUMO (Lumasiran) received approval from the European Commission (EC) for both pediatric and adult patients with…
According to a story from Fierce Pharma, the drug company Alnylam Pharmaceuticals has seen its investigational therapy lumasiran (marketed as Oxlumo) recently gain approval for use in the EU as…
According to a recent article in BioSpace, young children and infants who have been diagnosed with primary hyperoxaluria type 1 (PH1) are often faced with the use of gastrostomy…
According to a story from BioSpace, the biopharmaceutical company Dicerna Therapeutics, Inc. has announced recently that the US Food and Drug Administration (FDA) has granted Rare Pediatric Disease designation to…
In the past, there have been few effective treatments for patients with primary hyperoxaluria type 1 (PH1), a genetic disorder causing kidney damage and failure. But, according to News Medical,…
Molly Ouimet was three years old when she got her first kidney stone. Molly, who wasn't very vocal at the time, had a difficult time explaining the pain that she…
Copyscape score: 2% Alnylam has recently completed the third phase of their clinical trial of lumasiran, which was created for the treatment of primary hyperoxaluria type 1 (PH1). While they…
Big development for those in the primary hyperoxaluria type 1 (PH1) community! Alnylam Pharmaceuticals just announced that its Phase 3 study of lumasiran, an investigational therapy for the treatment of primary…
Alnylam Pharmaceuticals has developed and produced a cartoon series aimed at educating people -- especially young people -- about the pediatric rare disease primary hyperoxaluria type 1 (PH1). The campaign…
Alnylam Pharmaceuticals has been developing lumasiran as a potential treatment for primary hyperoxaluria type 1 (PH1). This investigation began with ILLUMINATE-A, a clinical trial whose results should be announced by…
Alnylam has provided updates about their programs for patients with primary hyperoxaluria type 1 (PH1), a rare condition that affects the kidneys and bladder. As part of their Alnylam Act® program,…
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.…
An Indian family found out their little girl, Parvathy, only three months old, had primary hyperoxaluria type 1. In simple terms, those words meant she was born without the enzyme…
According to QC Online, at six months old, Lorelei Lovelady was diagnosed with primary hyperoxaluria type 1 (PH1). Now, almost 9 months, the disease has caused her to need a new liver…
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