Quantcast
Last updated on April 17, 2014 at 11:05 EDT

Latest Alagille syndrome Stories

2013-11-18 08:28:41

Company receives first positive opinion in the European Union for Primary Sclerosing Cholangitis SAN DIEGO, Nov. 18, 2013 /PRNewswire/ -- Lumena Pharmaceuticals, a company developing oral therapeutics for rare liver diseases, today announced that it has received positive opinions for four Orphan Drug Designations by the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) for LUM001. The company's lead drug candidate, LUM001, received the first positive...

2013-11-05 08:34:31

Data Presented Today at Annual Meeting of the American Association for the Study of Liver Diseases in Washington DC Provides Additional Preclinical Validation for LUM001 Currently Being Evaluated in Phase II Studies in Multiple Cholestatic Liver Disease Populations SAN DIEGO, Nov. 5, 2013 /PRNewswire/ -- Lumena Pharmaceuticals, a company developing oral therapeutics for cholestatic liver diseases, today announced that inhibition of the apical sodium-dependent bile acid transporter...

2013-10-29 08:32:43

Novel therapeutic approach to be evaluated for its ability to reduce elevated bile acid levels and associated severe itching SAN DIEGO, Oct. 29, 2013 /PRNewswire/ -- Lumena Pharmaceuticals, a company developing oral therapeutics for rare liver diseases, today announced the dosing of the first patient in the CLARITY Phase II study of its drug candidate LUM001 in patients with primary biliary cirrhosis (PBC). LUM001 is being developed as a possible therapy for a number of cholestatic...

2013-09-26 08:27:28

First patient dosed in U.K. in Phase II IMAGO Study; Phase II ITCH Study to start in U.S. SAN DIEGO, Sept. 26, 2013 /PRNewswire/ -- Lumena Pharmaceuticals, a company developing oral therapeutics for rare liver diseases, today announced the initiation of a global clinical program to evaluate its drug candidate LUM001 in children with Alagille syndrome (ALGS). The first patient has been dosed in the IMAGO Phase II study being conducted in the U.K., and enrollment of pediatric...

2013-09-26 08:27:25

Company Pursuing Orphan Path to Development in Alagille Syndrome, Progressive Familial Intrahepatic Cholestasis, Primary Biliary Cirrhosis and Primary Sclerosing Cholangitis SAN DIEGO, Sept. 26, 2013 /PRNewswire/ -- Lumena Pharmaceuticals, a company developing oral therapeutics for rare liver diseases, today announced that it has been granted Orphan Drug Designation by the U.S. Food & Drug Administration (FDA) Office of Orphan Product Development for LUM001, the company's lead...

2013-06-06 23:01:33

Stories of Hope, Success and Progress Illustrate Need for Collaboration With Patients Horsham, Pa. (PRWEB) June 06, 2013 Utah Assistant Attorney General and spinal muscular atrophy survivor Steve Mikita and five other inspiring patient advocates will share stories of their tireless efforts to insert the patient voice at each step of the drug development process at the DIA 2013 49th Annual Meeting in Boston this month. The news media are invited to listen to the compelling stories of these...

Same Musicians Play Brand New Tune
2013-05-15 07:54:37

Stowers Institute for Medical Research A small ensemble of musicians can produce an infinite number of melodies, harmonies and rhythms. So too, do a handful of workhorse signaling pathways that interact to construct multiple structures that comprise the vertebrate body. In fact, crosstalk between two of those pathways–those governed by proteins known as Notch and BMP (for Bone Morphogenetic Protein) receptors–occurs over and over in processes as diverse as forming a tooth,...

2013-05-08 08:29:58

Funding to support clinical development of drug candidates to treat rare liver diseases SAN DIEGO, May 8, 2013 /PRNewswire/ -- Lumena Pharmaceuticals, a company developing oral therapeutics for rare liver diseases, today announced the company has secured $23 million in Series A financing. Investors include Pappas Ventures, RiverVest Venture Partners and Alta Partners. Founded in 2011 by Pappas Ventures, Lumena will primarily use the funding to advance the clinical development of...

2013-02-13 17:35:32

Researchers from the University of Pennsylvania School of Veterinary Medicine have discovered that a protein called Jagged-1 stimulates human stem cells to differentiate into bone-producing cells. This protein could help both human and animal patients heal from bone fractures faster and may form the basis of treatments for a rare metabolic condition called Alagille syndrome. The study, published in the journal Stem Cells, was authored by three members of Penn Vet´s departments of...

2012-07-23 22:20:40

GOTHENBURG, Sweden, July 24, 2012 /PRNewswire/ -- Albireo AB (Gothenburg, Sweden; "Albireo"), a biopharmaceutical company specializing in gastroenterology, today announced that the European Medicines Agency Committee for Orphan Medicinal Products (COMP) has issued a positive opinion on an application for orphan medicinal product status for the company's lead hepatology candidate, A4250, for the treatment of: - Primary Biliary Cirrhosis (PBC) -...