You can't be a pilot and a Cystinuric ;( Alvin Landrum lives in Carrollton, a small town nearly 40 miles west of Atlanta, Georgia. At 55, he sees a handful of physicians and specialists on a regularly basis, from primary care doctors to nephrologists to urologists, and takes 22 pills every day. Alvin is [...]
‘Pharma Bro’ Fraudster Martin Shkreli Freed from Prison Original Story Here https://www.nationalreview.com/news/pharma-bro-martin-shkreli-freed-from-prison/ Convicted pharmaceutical fraudster Martin Shkreli, known publicly as “Pharma Bro,” was released from prison on Wednesday, and will spend the rest of his sentence in a halfway house. Shkreli had been serving a seven-year sentence at the Federal Correctional Complex in Allentown, Pa., [...]
From: https://pubmed.ncbi.nlm.nih.gov/34982522/ Abstract Purpose of review: The aim of this study was to summarize recent findings in kidney gene therapy while proposing cystinuria as a model kidney disease target for genome engineering therapeutics. Recent findings: Despite the advances of gene therapy for treating diseases of other organs, the kidney lags behind. Kidney-targeted gene delivery remains [...]
Extracorporeal Shockwave Lithotripsy for Cystine Stones in Children: An Observational, Retrospective, Single-Center Analysis READ FULL STORY HERE Purpose: Cystinuria is a genetic disorder characterized by a defective reabsorption of cystine and dibasic amino acids leading to development of urinary tract calculi from childhood onward. Cystine lithiasis is known to be resistant to fragmentation. [...]
A natural compound designated as an orphan drug for cystinuria READ FULL STORY HERE A natural compound with antioxidant properties, L-ergothioneine, has been designated as an orphan drug for cystinuria treatment by the European Medicines Agency (EMA). Studies in a murine model of this rare disease have shown that L-ergothioneine could prevent the [...]
Researchers find potential biomarker for appearance of stones in cystinuria A new study has identified a possible biomarker for the appearance of cystine stones in the urinary tract in a murine model of cystinuria. The work, published in the journal Antioxidants , has been coordinated by Virginia Nunes, researcher at the Institut d'Investigació [...]
A natural compound designated as an orphan drug for cystinuria See Original Story HERE A natural compound with antioxidant properties, L-ergothioneine, has been designated as an orphan drug for cystinuria treatment by the European Medicines Agency (EMA). Studies in a murine model of this rare disease have shown that L-ergothioneine could prevent the formation [...]
Orsini Specialty Pharmacy Announces Access To Tiopronin For The Treatment Of Severe Homozygous Cystinuria ELK GROVE VILLAGE, Ill., July 22, 2021 /PRNewswire/ -- Orsini Specialty Pharmacy announced today that it is now dispensing tiopronin, the first available generic version, in the US, of THIOLA® (tiopronin). Tiopronin is a reducing and complexing thiol indicated, [...]
Advicenne Receives European Commission Approval to Market ADV7103 (Sibnayal™) and announces Leadership Changes to support Commercialisation ADV7103 becomes the first and only label-approved drug for the treatment of distal renal tubular acidosis (dRTA) in adults, adolescents and children aged one year and older Didier Laurens to join Advicenne as new CEO Resumption of trading [...]
My cystinuria and me. Hello, I am Nathalie, Venezuelan, married, 40 years old and clinical psychologist. My medical history began in 2004, at the age of 23 years with the following symptoms when I woke up one morning, I felt a very intense pain in the abdominal area that I almost fainted, and this [...]
