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Overview of Blueprint Medicines' kinase drug discovery strategy published in JCI

Overview of Blueprint Medicines' kinase drug discovery strategy published in JCI

Blueprint Medicines today announced that the Journal of Clinical Investigation published an overview of the Company's kinase drug discovery and development strategy. The publication underscores Blueprint Medicines' focus on identifying novel genomically defined disease drivers and the use of novel chemistry from its proprietary compound library to craft highly selective kinase inhibitors for new and difficult-to-drug kinase targets. [More]
University of Washington receives PPMD grant to continue analysis of spectrin-like repeats in dystrophin

University of Washington receives PPMD grant to continue analysis of spectrin-like repeats in dystrophin

Parent Project Muscular Dystrophy, a nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne) awarded University of Washington a $148,000 grant to continue the functional analysis of spectrin-like repeats in dystrophin. [More]
Amgen's Vectibix (panitumumab) receives EC approval for treatment patients with WT RAS mCRC

Amgen's Vectibix (panitumumab) receives EC approval for treatment patients with WT RAS mCRC

Amgen today announced that the European Commission approved a new use of Vectibix (panitumumab) as first-line treatment in combination with FOLFIRI for the treatment of adult patients with wild-type (WT) RAS metastatic colorectal cancer (mCRC). [More]
Duke researchers demonstrate genetic therapeutic technique to treat DMD patients

Duke researchers demonstrate genetic therapeutic technique to treat DMD patients

Duke researchers have demonstrated a genetic therapeutic technique that has the potential to treat more than half of the patients suffering from Duchenne Muscular Dystrophy (DMD). [More]
Rare and classic EGFR mutations have different impacts on NSCLC outcome

Rare and classic EGFR mutations have different impacts on NSCLC outcome

Certain rare epidermal growth factor receptor (EGFR) mutations are associated with tobacco smoking, worse prognosis and poor response to EGFR tyrosine kinase inhibitor (TKI) therapy compared to the more common "classical" EGFR mutations. [More]
EGFR–TKI response worse in NSCLC patients with rare EGFR mutations

EGFR–TKI response worse in NSCLC patients with rare EGFR mutations

In patients with non-small-cell lung cancer, rare epidermal growth factor receptor mutations are associated with a poorer response to EGFR–tyrosine kinase inhibitors compared with frequently occurring ones, according to a study published in Lung Cancer. [More]
Plasma EGFR mutation cfDNA predicts advanced lung cancer outcome

Plasma EGFR mutation cfDNA predicts advanced lung cancer outcome

Monitoring plasma cell-free DNA levels of epidermal growth factor receptor mutations may indicate prognosis for patients with lung adenocarcinoma after EGFR–tyrosine kinase inhibitor therapy, research suggests. [More]
Georgia Tech researchers unveil new version of genome annotation system

Georgia Tech researchers unveil new version of genome annotation system

Georgia Tech researchers, working with colleagues in the National Center for Biotechnology Information, have released a new version of a genome annotation system capable of analyzing more than 2,000 prokaryotic genomes per day, helping researchers accelerate prokaryotic genomics-based studies worldwide. [More]

Rare, complex EGFR mutations adversely affect EGFR–TKI treatment outcomes

Individuals with advanced non-small-cell lung cancer who have rare or complex epidermal growth factor receptor mutations have inferior outcomes in response to EGFR–tyrosine kinase inhibitor treatment compared with those with common mutations, research indicates. [More]
First-line dacomitinib may improve advanced NSCLC survival

First-line dacomitinib may improve advanced NSCLC survival

Preliminary research suggests that the second-generation tyrosine kinase inhibitor dacomitinib may improve progression-free survival in patients with advanced non-small-cell lung cancer with epidermal growth factor receptor mutations. [More]
Researchers show that iPS cells can be used to edit genetic mutations that cause DMD

Researchers show that iPS cells can be used to edit genetic mutations that cause DMD

Researchers at the Center for iPS Cell Research and Application, Kyoto University, show that induced pluripotent stem (iPS) cells can be used to correct genetic mutations that cause Duchenne muscular dystrophy (DMD). [More]
OGT's new CytoSure Medical Research Exome Array to be launched at ASHG 2014

OGT's new CytoSure Medical Research Exome Array to be launched at ASHG 2014

Oxford Gene Technology, The Molecular Genetics Company, will launch its CytoSure Medical Research Exome Array at the 64th Annual Meeting of the American Society of Human Genetics 2014, taking place in San Diego on October 18-22. [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]
Survival benefits with erlotinib plus bevacizumab in EGFR-Mutated NSCLC

Survival benefits with erlotinib plus bevacizumab in EGFR-Mutated NSCLC

Combining erlotinib with bevacizumab could prolong progression-free survival in patients being treated for epidermal growth factor receptor-mutated non-small-cell lung cancer, phase II trial results suggest. [More]
MU researchers make new breakthrough in spinal muscular atrophy drug

MU researchers make new breakthrough in spinal muscular atrophy drug

According to recent studies, approximately one out of every 40 individuals in the United States is a carrier of the gene responsible for spinal muscular atrophy (SMA), a neurodegenerative disease that causes muscles to weaken over time. [More]
Scientists identify genetic mutations in endometrioid endometrial carcinoma

Scientists identify genetic mutations in endometrioid endometrial carcinoma

Parents of twins often tell them apart through subtle differences such as facial expression, moles, voice tone and gait. Similarly, physicians treating women with endometrial cancer must be able to distinguish between different versions of this disease form that, on the surface, appear the same. [More]
Mutant allele frequency predicts lung cancer TKI response

Mutant allele frequency predicts lung cancer TKI response

Tyrosine kinase inhibitor sensitivity may be predicted by the frequency of a point mutation in the epidermal growth factor receptor gene, suggests a study of Japanese patients with advanced lung adenocarcinoma. [More]
Isoform: New protein offers novel therapeutic approach for patients with DMD

Isoform: New protein offers novel therapeutic approach for patients with DMD

Scientists have discovered a new form of dystrophin, a protein critical to normal muscle function, and identified the genetic mechanism responsible for its production. [More]

Co-targeting strategies suggested for EGFR-TKI resistance in NSCLC

Researchers have identified additional resistance mechanisms that could be targeted to improve the efficacy of irreversible epidermal growth factor receptor-tyrosine kinase inhibitors in patients with non-small-cell lung cancer harbouring both EGFR and T790M mutations. [More]
Collaboration classification useful for Japanese lung cancer patients

Collaboration classification useful for Japanese lung cancer patients

Epidermal growth factor receptor status and prognosis in Japanese patients can be predicted by a recently developed lung adenocarcinoma classification system, say researchers. [More]
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