Rare Disease Report


The US FDA has granted Palladio Biosciences Investigational New Drug (IND) clearance to proceed with a Phase 2 clinical trial of lixivaptan capsules in patients with autosomal dominant polycystic kidney disease (ADPKD).
Prometic Life Sciences is developing a potential treatment for plasminogen deficiency. We spoke with Pierre Laurin about that treatment.
The company will share data later this month regarding their Phase 2 clinical study assessing blisibimod in patients with IgA nephropathy.
24 of the 25 HLA-sensitized patients had normalized kidney function 6 months after their transplant.
After evaluating 262 patients given methylprednisolone, they stopped the study based on the safety profile that emerged.
Bernie Williams, former centerfielder and 4-time World Series champion, is teaming up with Boehringer Ingelheim to raise awareness of the idiopathic pulmonary fibrosis (IPF).
The "Define-FMD" study is comparing the genetic makeup of fibroblasts from FMD patients to those from non-FMD patients.
Kevin Meyers, MBBSc, of the Children's Hospital of Philadelphia (CHOP) talks about the incidence and symptoms of fibromuscular dysplasia (FMD) in children.
Tommy's transplanted kidney is, if not the longest, one of the longest functioning live donor kidneys in U.S. history.
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