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FDA lifts partial clinical hold that paused enrollment of new patients in tazemetosta clinical trials

Epizyme, Inc., a clinical-stage company developing novel epigenetic therapies, today announced the U.S. Food and Drug Administration has lifted the partial clinical hold that had paused U.S.-based enrollment of new patients in its tazemetostat clinical trials.

24 Sep 2018

SNMMI announces recipients of 2018-2020 Wagner-Torizuka Fellowship

The Society of Nuclear Medicine and Molecular Imaging is pleased to announce the recipients of the 2018-2020 SNMMI Wagner-Torizuka Fellowship.

24 Sep 2018

CHMP adopts positive opinion of Takeda’s ALUNBRIG for treatment of ALK+ non-small cell lung cancer

Takeda Pharmaceutical Company Limited today announced that the European Medicines Agency's Committee for Medicinal Products for Human Use has adopted a positive opinion, recommending the full approval of ALUNBRIG® as a monotherapy for the treatment of adult patients with anaplastic lymphoma kinase-positive advanced non-small cell lung cancer previously treated with crizotinib.

24 Sep 2018

Study sheds light on molecular mechanisms underlying progression of prion diseases

A new study has shed light on the mechanisms underlying the progression of prion diseases and identified a potential target for treatment.

21 Sep 2018

Study opens new avenues for treatment of Laing distal myopathy

New avenues are now being opened for future treatment of Laing distal myopathy, a rare disorder that causes muscles in the feet, hands and elsewhere to atrophy. In a study published in the journal PNAS, researchers have identified an enzyme with a clear link to how the disease develops.

21 Sep 2018

$CHMP grants positive opinion for VENCLYXTO plus rituximab for treating relapsed/refractory CLL$

CHMP grants positive opinion for VENCLYXTO plus rituximab for treating relapsed/refractory CLL

AbbVie, a research-based global biopharmaceutical company, announced today that the Committee for Medicinal Products for Human Use of the European Medicines Agency has granted a positive opinion for VENCLYXTO® in combination with rituximab for the treatment of patients with relapsed/refractory chronic lymphocytic leukemia who have received at least one prior therapy.

21 Sep 2018

New research finds that MHC-II molecules have more influence on tumors than MHC-I

Cancer immunotherapy -- efforts to boost a patient's own immune system, allowing it to better fight cancer cells on its own -- has shown great promise for some previously intractable cancers.

21 Sep 2018

Study could allow doctors to screen patients at risk from Aspergillus

Scientists at The University of Manchester have discovered a genetic mutation in humans linked to a 17-fold increase in the amount of dangerous fungal spores in the lungs.

20 Sep 2018

First U.S. patient treated with innovative gene therapy at Bascom Palmer Eye Institute

A Puerto Rican patient with X-linked retinitis pigmentosa (XLRP) is hoping to save his vision after an innovative gene therapy procedure at Bascom Palmer Eye Institute at the University of Miami Miller School of Medicine.

19 Sep 2018

Study reveals new therapeutic target for pediatric tumor-associated intractable epilepsy

Pediatric brain tumors are characterized by frequent complications due to intractable epilepsy compared to adult brain tumors. However, the genetic cause of refractory epilepsy in pediatric brain tumors has not been elucidated yet, and it is difficult to treat patients because the tumors do not respond to existing antiepileptic drugs and debilitate children's development.

19 Sep 2018

Alector announces initiation of Phase 1 trial of AL001 for treating frontotemporal dementia

Alector, a privately held biotechnology company pioneering immuno-neurology, a novel therapeutic approach for the treatment of neurodegeneration, today announced the initiation of its Phase 1 trial called INFRONT, which is evaluating AL001, the company's product candidate for the treatment of a genetically-defined sub-population of patients with frontotemporal dementia.

19 Sep 2018

Penn researchers find common thread linking almost all TNR expansion diseases

In a class of roughly 30 neurological disorders that includes ALS, Huntington's Disease and Fragile X Syndrome, the relevant mutant gene features sections of repeating base pair sequences known as short tandem repeats, or STRs. Healthy people have STRs of normal lengths distributed across their DNA.

19 Sep 2018

Research shows enlarged genotype-phenotype correlation for three-base pair deletion in NF1

International collaborative research led by Ludwine Messiaen, Ph.D., shows that while a three-base pair, in-frame deletion called p.Met992del in the NF1 gene has a mild phenotype for people with the genetic disorder neurofibromatosis type 1, or NF1, the mutation does cause complications.

18 Sep 2018

Brigham Genomic Medicine program unravels 30 medical mysteries

A table in a recently published paper tells the story of 30 families who have, sometimes after years of searching, finally received an answer about the condition that has plagued one or more family members.

18 Sep 2018

Promising gene therapy for 'day blind' sheep now safe for clinical trials in human patients

Back in 2009, a group of Israeli researchers identified a herd of Awassi sheep suffering from "day blindness". As its name implies, these sheep were blind during the day (in bright light) but could see at night, in low-light conditions.

17 Sep 2018

Salk research in yeast leads to serendipitous finding about hypomyelinating leukodystrophy

Studying the fundamental aspects of biology can sometimes lead to unexpected findings that directly relate to human disease. In one of the latest examples of scientific serendipity, researchers from the Salk Institute found that an important quality control mechanism in baker's yeast is closely connected to hypomyelinating leukodystrophy, a debilitating disease found in children.

17 Sep 2018

Twice-a-year MRI more effective than mammograms for women with genetic breast cancer risk

Getting magnetic resonance imaging scans twice a year instead of one annual mammogram is far more effective at detecting early breast cancers in young women with a high-risk genetic profile than mammograms alone, according to a research team based at the University of Chicago Medicine and the University of Washington, Seattle.

14 Sep 2018

New research sheds light on the causes of diabetes

The main cause of all forms of diabetes is pancreatic beta-cell dysfunction. Beta cells, found in the pancreatic islets, store and release insulin.

14 Sep 2018

Is ‘precision medicine’ the answer to cancer? Not precisely.

Facing incurable breast cancer at age 55, MaryAnne DiCanto put her faith in "precision medicine" — in which doctors try to match patients with drugs that target the genetic mutations in their tumors. She underwent repeated biopsies to identify therapies that might help.

13 Sep 2018

Study provides complete picture of HPV virus-head and neck cancer connections

Human papillomavirus is widely known to cause nearly all cases of cervical cancer. However, you might not know that HPV also causes 70 percent of oropharyngeal cancer, a subset of head and neck cancers that affect the mouth, tongue, and tonsils.

12 Sep 2018

Mutation News and Research

FDA lifts partial clinical hold that paused enrollment of new patients in tazemetosta clinical trials

SNMMI announces recipients of 2018-2020 Wagner-Torizuka Fellowship

CHMP adopts positive opinion of Takeda’s ALUNBRIG for treatment of ALK+ non-small cell lung cancer

Study sheds light on molecular mechanisms underlying progression of prion diseases

Study opens new avenues for treatment of Laing distal myopathy

CHMP grants positive opinion for VENCLYXTO plus rituximab for treating relapsed/refractory CLL

New research finds that MHC-II molecules have more influence on tumors than MHC-I

Study could allow doctors to screen patients at risk from Aspergillus

First U.S. patient treated with innovative gene therapy at Bascom Palmer Eye Institute

Study reveals new therapeutic target for pediatric tumor-associated intractable epilepsy

Alector announces initiation of Phase 1 trial of AL001 for treating frontotemporal dementia

Penn researchers find common thread linking almost all TNR expansion diseases

Research shows enlarged genotype-phenotype correlation for three-base pair deletion in NF1

Brigham Genomic Medicine program unravels 30 medical mysteries

Promising gene therapy for 'day blind' sheep now safe for clinical trials in human patients

Salk research in yeast leads to serendipitous finding about hypomyelinating leukodystrophy

Twice-a-year MRI more effective than mammograms for women with genetic breast cancer risk

New research sheds light on the causes of diabetes

Is ‘precision medicine’ the answer to cancer? Not precisely.

Study provides complete picture of HPV virus-head and neck cancer connections

Industry Focus eBook - Analytical Chemistry (2nd Edition) eBook

Improving healthcare sustainability through carbon data

Managing vibration in IVF laboratories

A New Approach to Preventing Bladder Cancer Recurrence

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