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Isis Pharmaceuticals begins ISIS-DMPK Rx clinical study in DM1 patients

Isis Pharmaceuticals begins ISIS-DMPK Rx clinical study in DM1 patients

Isis Pharmaceuticals, Inc. announced today that it has initiated a study for ISIS-DMPK Rx in patients with Myotonic Dystrophy Type 1 (DM1). DM1 is a rare genetic neuromuscular disease caused by the production of toxic dystrophia myotonica-protein kinase (DMPK) RNA in cells. ISIS-DMPKRx is specifically designed to reduce toxic DMPK RNA. [More]
Isis Pharmaceuticals announces initiation of ISIS-SMN Rx Phase 3 study in children with SMA

Isis Pharmaceuticals announces initiation of ISIS-SMN Rx Phase 3 study in children with SMA

Isis Pharmaceuticals, Inc. announced today the initiation of a pivotal Phase 3 study evaluating ISIS-SMNRx in approximately 120 non-ambulatory children with spinal muscular atrophy (SMA). [More]
Isis receives $10 million from Biogen Idec to initiate IND-supporting studies of ISIS-BIIB3 Rx

Isis receives $10 million from Biogen Idec to initiate IND-supporting studies of ISIS-BIIB3 Rx

Isis Pharmaceuticals, Inc. announced today that it has earned a $10 million milestone payment from Biogen Idec related to the initiation of investigational new drug (IND)-supporting studies of ISIS-BIIB3Rx. ISIS-BIIB3Rx is a recently identified development candidate designed to inhibit an undisclosed target to treat a neurodegenerative disease. [More]
Isis Pharmaceuticals provides update on Phase 2 clinical studies of ISIS-SMN Rx in children with SMA

Isis Pharmaceuticals provides update on Phase 2 clinical studies of ISIS-SMN Rx in children with SMA

Isis Pharmaceuticals, Inc. today provided an update on its ongoing open-label Phase 2 clinical studies of ISIS-SMN Rx in infants and children with spinal muscular atrophy at the 19th International World Muscle Society (WMS) Congress in Berlin, Germany. [More]
Research could help eventual treatment of degenerative muscle, brain diseases

Research could help eventual treatment of degenerative muscle, brain diseases

Our genetic information is stored in DNA, tiny strands of nucleic acid that contain instructions for the functioning of our bodies. To express this genetic data, our DNA is copied into RNA molecules, which then translate the instructions into proteins that perform tasks in our cells. [More]

New approach to turn diseased cells into unique manufacturing sites for treating muscular dystrophy

In a new study that could ultimately lead to many new medicines, scientists from the Florida campus of The Scripps Research Institute (TSRI) have adapted a chemical approach to turn diseased cells into unique manufacturing sites for molecules that can treat a form of muscular dystrophy. [More]
Isis Pharmaceuticals begins ISIS-DMPK Rx Phase 1 study for treatment of Myotonic Dystrophy Type 1

Isis Pharmaceuticals begins ISIS-DMPK Rx Phase 1 study for treatment of Myotonic Dystrophy Type 1

Isis Pharmaceuticals, Inc. announced today that it has initiated a Phase 1 study for ISIS-DMPKRx. Isis earned a $14 million milestone payment from Biogen Idec associated with this achievement. [More]
Isis Pharmaceuticals reports pro forma net operating loss of $22.6 million for Q1 2014

Isis Pharmaceuticals reports pro forma net operating loss of $22.6 million for Q1 2014

Isis Pharmaceuticals, Inc. today reported a pro forma net operating loss (NOL) of $22.6 million for the three months ended March 31, 2014 compared to pro forma operating income of $4.5 million for the same period in 2013. [More]
Researchers use RNA sequencing to look at early- and late-stage development of the heart

Researchers use RNA sequencing to look at early- and late-stage development of the heart

Most people think the development of the heart only happens in the womb, however the days and weeks following birth are full of cellular changes that play a role in the structure and function of the heart. Using mouse models, researchers at Baylor College of Medicine have now been able to categorize the alternative splicing (the process in which genes code proteins, determining their role) that takes place during these changes and what mechanisms they affect. [More]
U.Va. researchers named recipients of 2013 Hartwell Individual Biomedical Research Awards

U.Va. researchers named recipients of 2013 Hartwell Individual Biomedical Research Awards

University of Virginia neurologist Dr. Erin Pennock Foff, biologist Sarah Kucenas and biomedical engineer Shayn Peirce-Cotter have been named recipients of 2013 Hartwell Individual Biomedical Research Awards to benefit children of the United States. Each scientist will receive $100,000 in direct annual research support from The Hartwell Foundation for three years. [More]
Isis Pharmaceuticals provides update on ISIS-SMNRx Phase 2 study in infants with SMA

Isis Pharmaceuticals provides update on ISIS-SMNRx Phase 2 study in infants with SMA

Isis Pharmaceuticals, Inc. today provided an update on its ongoing open label, multiple dose Phase 2 study of ISIS-SMNRx in infants with spinal muscular atrophy (SMA). [More]

Scientists reveal atomic-level view of genetic defect that causes myotonic dystrophy type 2

Scientists from the Florida campus of The Scripps Research Institute have revealed an atomic-level view of a genetic defect that causes a form of muscular dystrophy, myotonic dystrophy type 2, and have used this information to design drug candidates with potential to counter those defects—and reverse the disease. [More]
Marina Biotech, Mirna modify license agreement regarding development of microRNA-based therapeutics

Marina Biotech, Mirna modify license agreement regarding development of microRNA-based therapeutics

Marina Biotech, Inc., a leading oligonucleotide-based drug discovery and development company, and Mirna Therapeutics, Inc., a privately-held biotechnology company pioneering microRNA replacement therapy for cancer, announced today that they have amended their license agreement regarding the development and commercialization of microRNA-based therapeutics utilizing Mirna's proprietary microRNAs and Marina Biotech's novel SMARTICLES liposomal delivery technology. [More]
Stem cell lines are ideal research tools for designing models to understand disease progression

Stem cell lines are ideal research tools for designing models to understand disease progression

Scientists from King's College London have announced that 16 human embryonic stem (hES) cell lines have been approved by the US National Institutes of Health (NIH) and placed on their Stem Cell Registry, making them freely available for federally-funded research in the USA. [More]
Researchers identify likely molecular pathway that causes untreatable neurodegenerative diseases

Researchers identify likely molecular pathway that causes untreatable neurodegenerative diseases

University of Adelaide researchers have identified a likely molecular pathway that causes a group of untreatable neurodegenerative diseases, including Huntington's disease and Lou Gehrig's disease. [More]
Scientists identify RNA regulator that plays critical role in proper functioning of hESCs

Scientists identify RNA regulator that plays critical role in proper functioning of hESCs

Scientists at A*STAR's Genome Institute of Singapore, in collaboration with their counterparts from Canada, Hong Kong and US, have discovered a protein mediator SON plays a critical role in the health and proper functioning of human embryonic stem cells. This finding was reported on 8th September 2013 in the advanced online issue of the prestigious science journal Nature Cell Biology. [More]
Biogen Idec And Isis Ink Fourth Deal In Two Years

Biogen Idec And Isis Ink Fourth Deal In Two Years

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Novel way to dramatically raise potency of drug candidates targeting RNA

Novel way to dramatically raise potency of drug candidates targeting RNA

Scientists from the Jupiter campus of The Scripps Research Institute (TSRI) have shown a novel way to dramatically raise the potency of drug candidates targeting RNA, resulting in a 2,500-fold improvement in potency and significantly increasing their potential as therapeutic agents. [More]
TSRI scientists identify small molecules to control genetic defect responsible for muscular dystrophy

TSRI scientists identify small molecules to control genetic defect responsible for muscular dystrophy

For the first time, scientists from the Florida campus of The Scripps Research Institute have identified small molecules that allow for complete control over a genetic defect responsible for the most common adult onset form of muscular dystrophy. [More]
New hope for patients with myotonic dystrophy

New hope for patients with myotonic dystrophy

There's hope for patients with myotonic dystrophy. A new small molecule developed by researchers at the University of Illinois has been shown to break up the protein-RNA clusters that cause the disease in living human cells, an important first step toward developing a pharmaceutical treatment for the as-yet untreatable disease. [More]