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Johns Hopkins researchers discover role of TDP-43 protein in autopsy brain cells of patients with ALS

Autopsies of nearly every patient with the lethal neurodegenerative disorder amyotrophic lateral sclerosis (ALS), and many with frontotemporal dementia (FTD), show pathologists telltale clumps of a protein called TDP-43. Now, working with mouse and human cells, Johns Hopkins researchers report they have discovered the normal role of TDP-43 in cells and why its abnormal accumulation may cause disease.

7 Aug 2015

Rare BCR-ABL fusions highlighted in CML

Chronic myeloid leukaemia patients with rare BCR-ABL fusions may be missed by quantitative polymerase chain reaction, research suggests.

24 Jul 2015

OGT’s popular ESHG workshop free to view online

Oxford Gene Technology (OGT), The Molecular Genetics Company, has made its European Human Genetics (ESHG) Conference workshop freely available to view online.

From Oxford Gene Technology 16 Jul 2015

FDA accepts Blueprint Medicines' IND applications to start Phase 1 clinical trials for two drug candidates

Blueprint Medicines today announced that the U.S. Food and Drug Administration accepted the Company's Investigational New Drug (IND) applications to begin Phase 1 clinical trials for its two lead drug candidates: BLU-554 for the treatment of advanced hepatocellular carcinoma (HCC) and cholangiocarcinoma, and BLU-285 for unresectable, treatment-resistant gastrointestinal stromal tumor (GIST).

13 Jul 2015

$Phase 3 study shows Vectibix meets primary endpoint in patients with chemorefractory wild-type KRAS mCRC$

Phase 3 study shows Vectibix meets primary endpoint in patients with chemorefractory wild-type KRAS mCRC

Amgen today announced that a Phase 3 study evaluating Vectibix (panitumumab) and best supportive care (BSC) met its primary endpoint, demonstrating a statistically significant improvement in overall survival (OS) in patients with chemorefractory wild-type KRAS (exon 2) metastatic colorectal cancer (mCRC) compared to those patients treated with BSC alone.

19 Jun 2015

NCCN ORP receives $2-million grant from Boehringer Ingelheim to facilitate studies of afatinib in NSCLC

The National Comprehensive Cancer Network Oncology Research Program (ORP) has been awarded a $2-million grant from Boehringer Ingelheim Pharmaceuticals, Inc. to develop a program to evaluate scientifically targeted combination approaches with afatinib in the treatment of non-small cell lung cancer (NSCLC).

12 Jun 2015

OGT launches new arrays for developmental disorders at ESHG

Oxford Gene Technology (OGT), The Molecular Genetics Company, is today launching a new range of arrays at the European Society of Human Genetics (ESHG) Conference in Glasgow, UK, that are dedicated to elucidating the underlying causes of developmental delay.

From Oxford Gene Technology 10 Jun 2015

Boehringer Ingelheim presents afatinib LUX-Lung 8 trial data at ASCO 2015

2 Jun 2015

Boehringer announces LUX-Lung 8 data that compares efficacy of afatinib, erlotinib in patients with advanced SCC

Boehringer Ingelheim today announced overall survival (OS) results from the LUX-Lung 8 trial (NCT01523587) that directly compared the efficacy and safety of two EGFR-directed treatments, afatinib and erlotinib, in patients with advanced squamous cell carcinoma (SCC) of the lung, progressing after treatment with first-line chemotherapy. Treatment with afatinib significantly reduced the risk of death by 19%, extending the survival of patients to a median of 7.9 months compared to 6.8 months on erlotinib.

1 Jun 2015

Clinical utility of Caris Molecular Intelligence in five studies to be presented at ASCO 2015

Caris Life Sciences, a leading biotechnology company focused on fulfilling the promise of precision medicine, today announced the presentation of data from five studies that demonstrate the clinical utility of Caris Molecular Intelligence®, the company's panomic comprehensive tumor profiling service, in helping to identify targeted treatment options for patients with rare and/or aggressive tumors.

1 Jun 2015

Mirati Therapeutics presents preliminary results of MGCD265 tyrosine kinase inhibitor at ASCO 2015

Mirati Therapeutics, Inc. today presented data that demonstrated preliminary evidence of clinical activity from its investigational targeted tyrosine kinase inhibitor candidate, MGCD265, as part of the developmental therapeutics category at the 2015 American Society of Clinical Oncology (ASCO) Annual Meeting being held in Chicago from May 29-June 2, 2015.

1 Jun 2015

Multiplatform biomarker analysis reveals new therapeutic options for squamous cell anal carcinomas

While squamous cell anal carcinomas are rare, representing only about 2% of digestive-system cancer diagnoses, these cancers, which are associated with the human papillomavirus (HPV), sometimes prove very difficult to treat, recurring or developing metastases following standard treatment.

30 May 2015

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.

11 May 2015

Positive data from two studies analyzing Good Start's test for inherited diseases presented at ACOG 2015

Good Start Genetics, Inc., a commercial-stage molecular genetics information company, today presented data from two studies analyzing detection rates among patients at in vitro fertilization (IVF) clinics who underwent carrier screening for inherited diseases. The studies were presented at the 2015 Annual Clinical and Scientific Meeting of The American College of Obstetricians and Gynecologists being held in San Francisco, Calif.

7 May 2015

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.

20 Apr 2015

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).

6 Apr 2015

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).

19 Feb 2015

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.

12 Feb 2015

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.

28 Jan 2015

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.

28 Jan 2015

Exon News and Research

Johns Hopkins researchers discover role of TDP-43 protein in autopsy brain cells of patients with ALS

Rare BCR-ABL fusions highlighted in CML

OGT’s popular ESHG workshop free to view online

FDA accepts Blueprint Medicines' IND applications to start Phase 1 clinical trials for two drug candidates

Phase 3 study shows Vectibix meets primary endpoint in patients with chemorefractory wild-type KRAS mCRC

NCCN ORP receives $2-million grant from Boehringer Ingelheim to facilitate studies of afatinib in NSCLC

OGT launches new arrays for developmental disorders at ESHG

Boehringer Ingelheim presents afatinib LUX-Lung 8 trial data at ASCO 2015

Boehringer announces LUX-Lung 8 data that compares efficacy of afatinib, erlotinib in patients with advanced SCC

Clinical utility of Caris Molecular Intelligence in five studies to be presented at ASCO 2015

Mirati Therapeutics presents preliminary results of MGCD265 tyrosine kinase inhibitor at ASCO 2015

Multiplatform biomarker analysis reveals new therapeutic options for squamous cell anal carcinomas

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

Positive data from two studies analyzing Good Start's test for inherited diseases presented at ACOG 2015

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

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

Duke researchers demonstrate genetic therapeutic technique to treat DMD patients

Rare and classic EGFR mutations have different impacts on NSCLC outcome

Plasma EGFR mutation cfDNA predicts advanced lung cancer outcome

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

Achieving and maintaining ISO/IEC 27001 and BS 10012 certifications

Expanding research and clinical options for children with cancer

Examining how pain could play a direct protective role in the gut

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