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Research in the News

Acceleron Announces Plans to Initiate a Phase 2 Trial of ACE-083

Acceleron Announces Plans to Initiate a Phase 2 Trial of ACE-083

- Second Phase 2 study for ACE-083 expected to begin 2H 2017 Acceleron Pharma Inc, a clinical stage biopharmaceutical company focused on the discovery, development and commercialization of innovative therapeutics to treat serious and rare diseases, today announced that in the second half of 2017 it plans to initiate a Phase 2 clinical trial of ACE-083, the Company's locally acting muscle agent, Read more...

New therapeutic targets identified in Charcot-Marie-Tooth disease

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Research conducted at the Institute of Neurology of University College London (UCL) and just published in the Proceedings of the National Academy of Sciences USA has identified a set of novel therapeutic targets for a subtype of Charcot-Marie-Tooth disease (CMT), a human condition that currently has no effective treatments. Surprisingly, the scientists also uncovered a previously unappreciated devRead more...

Understanding cell membrane changes

Understanding cell membrane changes

Understanding cell membrane changes in centronuclear myopathy and Charcot-Marie-Tooth disease Dr Andrew Shevchuk and his team from Imperial College London will investigate how mutations associated with some types of centronuclear myopathy and Charcot-Marie-Tooth disease (CMT) affect an essential cell process called endocytosis that allows cells to take up nutrients and replenish their cell membraRead more...

BioPontis Alliance for Rare Diseases

BioPontis Alliance for Rare Diseases

BioPontis Alliance for Rare Diseases today announces receipt of a large three year donation from the prestigious Baillet Latour Fund (BLF) in Belgium. BioPontis Alliance has the mission to translate biomedical research into therapies for rare diseases, and underwent rigorous vetting from BLF's high level Board and medical advisors. The funding is offered to BioPontis as part of BLF's commitment toRead more...

MRC Centre-BRC MRI Biomarker Collaboration in Lancet Neurology:

MRC Centre-BRC MRI Biomarker Collaboration in Lancet Neurology:

Important implications for experimental trials in Neuromuscular Diseases 9 November 2015 - http://www.ucl.ac.uk/ion/articles/news/20151106 A UCL Institute of Neurology study published in Lancet Neurology this week provides important prospective evidence that quantitative MRI can reliably and sensitively track disease progression in Neuromuscular Diseases. The research shows that tRead more...

New discovery at CING could benefit those with inherited neuropathy

New discovery at CING could benefit those with inherited neuropathy

A DISCOVERY by scientists at the Institute of Neurology and Genetics (CING) in Nicosia may open the way for gene therapy for to benefit patients with inherited neuropathy, also known as Charcot-Marie-Tooth Disease, according to a press release issued on Tuesday. Following 15 years of intense research to understand the cause and to develop experimental models for hereditary neuropathies such as Read more...

Exome Sequencing of Peripheral Neuropathy Patients IDs Candidate Genes

Exome Sequencing of Peripheral Neuropathy Patients IDs Candidate Genes

NEW YORK (GenomeWeb) – Through exome sequencing, researchers from Baylor College of Medicine and elsewhere identified apparent causal mutations and an increased burden of rare variants for patients with Charcot-Marie-Tooth-like peripheral neuropathies that had eluded diagnosis. CMT is a hereditary peripheral neuropathy with two major clinical types as well as other less-common forms that haRead more...

First IVF baby born after new technique to eliminate genetic disease

First IVF baby born after new technique to eliminate genetic disease

The first baby in Europe has been born following a new IVF-based technique developed to prevent the inheritance of genetic disease. The screening technique, called karyomapping, is a type of PGD (preimplantation genetic diagnosis) procedure. Current PGD methods are developed on a case-by-case basis to test embryos for the presence of a specific mutation found in one or both of the parenRead more...

New treatment strategy for patients with CMT disease on the horizon

New treatment strategy for patients with CMT disease on the horizon

Neuroscientists at UB's Hunter James Kelly Research Institute show how turning down synthesis of a protein improves nerve, muscle function in common neuropathy A potential new treatment strategy for patients with Charcot-Marie-Tooth disease is on the horizon, thanks to research by neuroscientists now at the University at Buffalo's Hunter James Kelly Research Institute and their colleagues in Read more...

New Screening Technologies Enable Identification of Potential Treatment Target for Neurological Disorder

New Screening Technologies Enable Identification of Potential Treatment Target for Neurological Disorder

Bethesda MD (Scicasts) — Improving scientific methods and tools is a key part of the NCATS approach to solving translational problems on a system-wide level, with the goal of delivering more treatments to more patients more quickly. A recent advance in this area — made possible through a project collaboration involving NCATS experts, academic researchers, a patient advocacy group and a pharmaceutiRead more...

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