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Latest Spliceosome Stories

2012-03-20 02:24:58

NEWTON, Mass., March 20, 2012 /PRNewswire/ -- Halo Therapeutics, LLC, a clinical-stage biopharmaceutical company developing novel therapeutics for rare fibrotic diseases, announced today that the TREAT-NMD Advisory Committee on Therapeutics (TACT) has reviewed HT-100, Halo's drug candidate for Duchenne muscular dystrophy (DMD), and believes the compound has potential based on the proposed mechanism. TREAT-NMD is a global network dedicated to expediting the delivery of promising...

2012-03-19 19:21:57

Investigators at Nationwide Children's Hospital, working with the DNA Sequencing Core Facility at the University of Utah, have developed an approach to newborn screening (NBS) for the life-threatening genetic disorder, Duchenne muscular dystrophy (DMD) and potentially other muscular dystrophies. As a model for NBS, the approach published online in January in the Annals of Neurology provides evidence that this approach could be implemented if approved by regulatory bodies at a state level or...

2012-03-02 13:00:00

Media integration and events community kicks off voting for awards contest representing the top influencers in the SMB channel. Redmond, WA (PRWEB) March 02, 2012 SMB Nation announced today at its MVP Nation event, that it has officially opened voting for its SMB 150, representing the top 150 influencers in the SMB channel. In addition, for the first time, the media integration and events community, in conjunction with SMB Technology Network (SMBTN), will be holding a live Gala Event on May...

2012-02-27 10:53:00

Parent Project Muscular Dystrophy Holds 13th Annual Advocacy Conference WASHINGTON, Feb. 27, 2012 /PRNewswire-USNewswire/ -- Nearly 80 parents, grandparents, and relatives of boys with the most common form of muscular dystrophy will be on Capitol Hill today and tomorrow urging Members of Congress to support critical research and patient care initiatives. (Logo: http://photos.prnewswire.com/prnh/20100119/DC39975LOGO ) Advocates from 23 states are participating in the...

2012-02-21 11:00:00

Grant Funds Work of Tivorsan Pharmaceuticals' Team to Bring Recombinant Biglycan to the Clinic for the Possible Treatment of Duchenne Muscular Dystrophy HACKENSACK, N.J., Feb. 21, 2012 /PRNewswire-USNewswire/ -- Parent Project Muscular Dystrophy (PPMD) announced today that it will award Tivorsan Pharmaceuticals (Tivorsan) a $500,000 grant to develop the company's biglycan therapeutic candidate for Duchenne muscular dystrophy. (Logo:...

2012-02-16 18:17:54

Understanding how RNA binding proteins control the genetic splicing code is fundamental to human biology and disease — much like editing film can change a movie scene. Abnormal variations in splicing are often implicated in cancer and genetic neurodegenerative disorders. In a step toward deciphering the "splicing code" of the human genome, researchers at the University of California, San Diego School of Medicine have comprehensively analyzed six of the more highly expressed RNA...

2012-01-19 14:55:49

Mutation in 1 copy of U2 snRNA lead to movement problems and early neuron death in mice A Jackson Laboratory research team led by Professor and Howard Hughes Medical Investigator Susan Ackerman, Ph.D., has discovered a defect in the RNA splicing process in neurons that may contribute to neurological disease. The researchers found that a mutation in just one of the many copies of a gene known as U2 snRNAs, which is involved in the intricate processing of protein-encoding RNAs, causes...

2012-01-05 02:00:00

TUCSON, Ariz. and PROVIDENCE, R.I., Jan. 5, 2012 /PRNewswire-USNewswire/ -- The Muscular Dystrophy Association (MDA) today announced it has awarded $1.0 million to Tivorsan Pharmaceuticals to help speed pre-clinical work vital to a filing of an Investigational New Drug (IND) application with the U.S. Food and Drug Administration (FDA) for a recombinant humanized form of biglycan as a potential muscular dystrophy treatment. The new funding to the Providence-based biotechnology firm comes from...

2012-01-04 07:00:00

NEWTON, Mass., Jan. 4, 2012 /PRNewswire/ -- Halo Therapeutics, LLC, a clinical-stage biopharmaceutical company developing novel therapeutics for rare fibrotic diseases, today announced that the United States Food and Drug Administration (FDA) granted orphan drug designation for HT-100 for treatment of Duchenne muscular dystrophy (DMD). DMD is a progressive and fatal neuromuscular disorder, which afflicts approximately 1 in 3,500 boys worldwide. HT-100, also known as halofuginone, is an orally...

2011-12-19 11:22:00

TUCSON, Ariz., Dec. 19, 2011 /PRNewswire-USNewswire/ -- The Muscular Dystrophy Association (MDA) has awarded $750,000 to Summit PLC, a UK biotechnology company, to continue development of an experimental drug that may become a viable treatment for boys with Duchenne muscular dystrophy (DMD). The funding comes from MDA Venture Philanthropy (MVP), a part of MDA's translational research program. DMD is one of nine types of muscular dystrophy, a group of genetic, degenerative diseases primarily...