Nephropathic Cystinosis News and Research

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UB research identifies key mechanism in infantile cystinosis

University at Buffalo research has identified how a misstep in the genesis of a key component of the kidney causes infantile cystinosis, a rare disease that significantly shortens the lifespan of patients.

11 Dec 2023

Insilico Medicine and MIKADO team up to find new treatments for cystinosis

A team led by researchers at the Mechanisms of Inherited Kidney Disorders (MIKADO) group at the University of Zurich (Zurich, Switzerland) has used Insilico Medicine's generative artificial intelligence (AI) target discovery engine, PandaOmics, to identify actionable drug targets for the lysosomal storage disease cystinosis and to validate them in preclinical models of the disease.

14 Jul 2023

Cysteamine inhibits SARS-CoV-2 variants of concern in vitro

Researchers in the United States have shown that a compound currently used to treat cystinosis exhibits broad antiviral activity against variants of severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) – the agent that causes coronavirus disease 2019 (COVID-19).

5 Oct 2021

Existing drug for rare kidney disease could benefit patients with mitochondrial disorders

New preclinical findings from extensive cell and animal studies suggest that a drug already used for a rare kidney disease could benefit patients with some mitochondrial disorders--complex conditions with severe energy deficiency for which no proven effective treatments exist.

27 Feb 2019

Cystinosis Research Foundation granted $2.15M in 2013 to researchers investigating cure for cystinosis

The Cystinosis Research Foundation awarded $2,153,048 in 2013 to researchers investigating better treatments and a cure for cystinosis, a rare metabolic and genetic disease that afflicts about 500 children and young adults in the United States and 2,000 worldwide.

19 Feb 2014

Raptor reopens enrollment in DR Cysteamine Phase 3 trial to treat patients with nephropathic cystinosis

Raptor Pharmaceutical Corp., today announced it has reopened enrollment in its Phase 3 clinical trial of its proprietary delayed-release oral formulation of cysteamine bitartrate in patients with nephropathic cystinosis.

15 Feb 2011

Raptor completes enrollment in DR Cysteamine Phase 3 clinical trial in cystinosis

Raptor Pharmaceutical Corp., today announced it has completed enrollment in its Phase 3 clinical trial of its proprietary delayed-release oral formulation of cysteamine bitartrate ("DR Cysteamine") in patients with nephropathic cystinosis ("cystinosis").

24 Jan 2011

Raptor Pharmaceutical updates on cysteamine bitartrate Phase 3 clinical trial in cystinosis patients

Raptor Pharmaceutical Corp.,provides updates on timelines related to its clinical programs including the pivotal Phase 3 clinical trial of its proprietary delayed-release oral formulation of cysteamine bitartrate ("DR Cysteamine") in patients with nephropathic cystinosis ("cystinosis").

24 Aug 2010

Raptor initiates enrollment in pivotal Phase 3 clinical trial of DR Cysteamine for nephropathic cystinosis

Raptor Pharmaceutical Corp. announced that it has enrolled the first patient in a pivotal Phase 3 clinical trial of its proprietary delayed-release oral formulation of cysteamine bitartrate ("DR Cysteamine") in patients with nephropathic cystinosis ("cystinosis").

28 Jun 2010

Raptor Pharmaceutical acquires exclusive license to intellectual property for Huntington's Disease treatment

Raptor Pharmaceutical Corp., announced that the Company has acquired an exclusive worldwide license to intellectual property related to the potential treatment of Huntington's Disease from the Weizmann Institute of Science in Israel and Niigata University in Japan. In addition, Raptor has added Professor Lawrence Steinman, M.D., an inventor on the Weizmann patent, to its Advisory Board.

2 Jun 2010

Raptor Pharmaceutical to present DR Cysteamine trial data at Lysosomal Disease Network Symposium

Raptor Pharmaceutical Corp., today announced the presentation of cystinosis data at the Annual Lysosomal Disease Network WORLD Symposium 2010, being held February 10-12 in Miami, Florida. Biomarker data from a Phase IIb pilot study of Raptor's proprietary delayed-release cysteamine bitartrate ("DR Cysteamine") will be the subject of a poster titled, "Correlation of Plasma Cysteamine and WBC Cystine Levels at Steady State in Patients Treated with Cysteamine Bitartrate." The results will also be published in an upcoming issue of the journal Molecular Genetics and Metabolism.

10 Feb 2010

Results from Phase 2a clinical trial of Raptor Pharmaceutical's cysteamine bitartrate published

Raptor Pharmaceutical Corp. ("Raptor" or the "Company"), today announced the publication of results from a Phase 2a clinical trial of a prototype formulation of its proprietary delayed-release cysteamine bitartrate ("DR Cysteamine") in patients with nephropathic cystinosis ("cystinosis").

11 Jan 2010

CRF awards $1.75 million in 11 new grants to researchers to find a cure for cystinosis

The Cystinosis Research Foundation of Irvine, Calif., announced it awarded $1.75 million in 11 new grants to researchers in the United States, Canada, Belgium and Italy who are working to find a cure for cystinosis, a rare, deadly metabolic and genetic disease that afflicts about 500 children and young adults in the U.S. and 2,000 worldwide.

7 Jan 2010

Raptor Pharmaceutical announces Phase 2b results of DR Cysteamine trial

Raptor Pharmaceutical Corp., today announced results from a Phase 2b clinical trial of its proprietary delayed-release cysteamine bitartrate ("DR Cysteamine") in patients with nephropathic cystinosis ("cystinosis"). The trial, conducted at the University of California, San Diego ("UCSD") General Clinical Research Center, evaluated safety, tolerability, pharmacokinetics and pharmacodynamics of a single dose of DR Cysteamine in nine cystinosis patients.

23 Nov 2009

Raptor and TorreyPines Therapeutics announce merger completion

Raptor Pharmaceuticals Corp. ("Raptor") (OTC Bulletin Board: RPTP) and TorreyPines Therapeutics, Inc. ("TorreyPines") (Nasdaq: TPTX) announced today the completion of their merger.

1 Oct 2009

Raptor Pharmaceuticals initiates phase IIa clinical study for Convivia

Raptor Pharmaceuticals Corp. has announced that its clinical development subsidiary, Bennu Pharmaceuticals Inc. , has initiated the Company's first clinical-stage program after dosing the first cohort in a Phase IIa study of oral 4-methylpyrazole ("4-MP") in subjects with ALDH2 deficiency, or ethanol intolerance, as the initial development stage of its Convivia program.

21 Apr 2008

Changes in the names of medicines, healthcare professionals to be vigilant against medication errors

Healthcare professionals should be aware that alphabetised lists of medicines may have changed and should be careful when selecting medicines to avoid medication errors.

30 Apr 2004