pharmaphorum3d
FDA approves Alnylam's ultra-rare disease drug Oxlumo
Patients with PH1 produce far too much oxalate, a substance consumed in food and produced by the body. This combines with calcium to cause kidney stones and deposits in the kidney, which can lead ...
Business Wire4d
PreventionGenetics Assumes the Management of Sponsored Genetic Testing Program for Three Rare Genetic Diseases: Acute Hepatic Porphyria, Primary Hyperoxaluria Type 1 ...
PH1 is a potentially life-threatening progressive genetic disease characterized by the accumulation of oxalate in the kidney due to an enzyme deficiency in the liver. Calcium oxalate crystals are ...
Morningstar20d
YolTech Therapeutics Announces Positive Clinical Data for YOLT-203, an In Vivo Gene Editing Therapy for Primary Hyperoxaluria Type 1 (PH1)
PH1, the most common subtype, is triggered by AGXT gene mutations and typically manifests in childhood. Patients often present with kidney stones, nephrocalcinosis, renal insufficiency ...
Yahoo Finance20d
Success for YolTech’s hyperoxaluria in vivo gene therapy in early-stage trial
The condition typically manifests in childhood with symptoms such as kidney stones and blood in urine ... levels through to week 16, a marker of PH1. At the same time, there were no reported ...
Hosted on MSN12d
How early detection could save lives in Primary Hyperoxaluria Type 1
Primary hyperoxaluria type 1 (PH1) is a metabolic stone disease that, if left undiagnosed, can have devastating consequences, including progressive kidney decline and end-stage kidney disease (ESKD).
Pharmabiz19d
YolTech Therapeutics reports positive clinical data for YOLT-203, an in vivo gene editing therapy for primary hyperoxaluria type 1
YolTech Therapeutics reports positive clinical data for YOLT-203, an in vivo gene editing therapy for primary hyperoxaluria type 1: Shanghai, China Wednesday, February 26, 2025, 1 ...