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New gene therapy trial for X-linked myotubular myopathy offers hope to children

Ann & Robert H. Lurie Children's Hospital of Chicago is one of the few centers participating in ASPIRO, an international Phase 1/2 clinical trial of a gene therapy product called AT132 for X-linked myotubular myopathy – a rare disease characterized by severe muscle weakness, breathing difficulty and early death.

8 Nov 2017

Major labeling inaccuracies in cannabidiol extract products

There is a growing demand among consumers for marijuana containing products supposedly providing several health benefits in a range od diseases and conditions. This has spawned thousands of websites that are selling these products online. Many of these contain mislabeled cannabidiol (CBD) or a compound found in cannabis or marijuana finds a new study.

7 Nov 2017

Study assesses representation of women and minorities in clinical drug trials

A new study to assess the effects of U.S.. Food and Drug Administration (FDA) regulations and guidance, intended to encourage greater inclusion of women and minorities in clinical drug trials, has shown appropriate levels of female participation based on the estimated sex ratio of people affected by a particular disease.

7 Nov 2017

Mislabeling of cannabidiol products poses safety and efficacy issues for consumers

Growing consumer demand for cannabis-related products purported to have medicinal benefits has created an environment where people are using mislabeled cannabidiol products sold online.

7 Nov 2017

SBP researchers identify new protective function for brain protein genetically linked to Alzheimer's

Researchers at Sanford Burnham Prebys Medical Discovery Institute have identified a new protective function for a brain protein genetically linked to Alzheimer's. The findings, published in the Journal of Experimental Medicine, could inform novel treatment strategies.

7 Nov 2017

FDA approves first targeted cancer treatment based on basket study

The US Food and Drug Administration has announced that it has approved the drug vemurafenib for the treatment of patients with BRAF V600-mutant Erdheim-Chester disease (ECD).

7 Nov 2017

FDA grants approval to first treatment for rare blood cancer

The U.S. Food and Drug Administration today expanded the approval of Zelboraf (vemurafenib) to include the treatment of certain adult patients with Erdheim-Chester Disease (ECD), a rare cancer of the blood.

7 Nov 2017

FDA approves new drug Zelboraf for Erdheim-Chester disease

The United States Food and Drugs Administration (FDA) has approved Zelboraf (vemurafenib), a drug for the treatment of adults with a are type of blood cancer called Erdheim-Chester Disease or ECD.

6 Nov 2017

Sleepless nights lead to sluggish brain activity

A new study has shown that some brain cells actually slow down if a person has not got adequate sleep at night. This leads to periods of mental lapses. Researchers of University of California, Los Angeles (UCLA) and Tel Aviv University have proven this in their published study that appeared in the journal Nature Medicine online this Monday (6th of November 2017).

6 Nov 2017

Single dose of measles vaccine not sufficient to protect children against disease in Bangladesh

Research in Bangladesh has found that a quarter of children need more than single doses of the measles vaccine to be protected against the disease. This research supports the country's recent move to a double-dose approach.

6 Nov 2017

Trial indicates blood-plasma infusions for Alzheimer’s patients are safe and may be effective

Researchers from Stanford University School of Medicine have found that giving blood plasma infusions taken from young donors to people with mild-to-moderate Alzheimer’s disease is not only safe and feasible, but may improve recipients’ functional ability and capacity to perform daily tasks.

6 Nov 2017

Digital health coaching program improves well-being, decreases symptoms for RA patients

A 12-week, digital health coaching program with personalized support from a dedicated health coach improved well-being and decreased symptoms for participants with rheumatoid arthritis, according to new research findings presented this week at the 2017 ACR/ARHP Annual Meeting in San Diego.

6 Nov 2017

Many patients with knee, hip and spine osteoarthritis use opioids to manage chronic pain

A large percentage of patients with end-stage knee, hip and spine osteoarthritis use opioids to manage their chronic pain, especially those who are younger or have symptoms of depression, according to new research findings presented this week at the 2017 ACR/ARHP Annual Meeting in San Diego.

6 Nov 2017

Internet-based educational program improves health-related quality of life for teens with JIA

An internet-based health education and self-management program that offers monthly calls with health coaches improved health-related quality of life for participating teens with juvenile idiopathic arthritis, according to new research findings presented this week at the 2017 ACR/ARHP Annual Meeting in San Diego.

6 Nov 2017

Low-energy ESWT shows promise for managing digital ulcers in patients with systemic sclerosis

Extracorporeal shock-wave therapy at low energy levels showed promise as a new treatment for digital ulcers in patients with systemic sclerosis, or scleroderma, according to new research findings presented this week at the 2017 ACR/ARHP Annual Meeting in San Diego.

6 Nov 2017

Researchers develop new drug screening method for rapid, accurate identification of fentanyl

Researchers at McMaster University have developed a new drug screening technique that could lead to the rapid and accurate identification of fentanyl, as well as a vast number of other drugs of abuse, which up until now have been difficult to detect by traditional urine tests.

3 Nov 2017

ACADIA Pharmaceuticals announces results from Phase II study of antipsychotic in Alzheimer's disease psychosis

ACADIA Pharmaceuticals Inc., a biopharmaceutical company focused on the development and commercialization of innovative medicines to address unmet medical needs in central nervous system (CNS) disorders, today announced the presentation of data from the Phase II -019 Study of pimavanserin in Alzheimer's disease psychosis at the 10th Clinical Trials on Alzheimer's Disease meeting in Boston.

3 Nov 2017

Diabetes awareness month: Mount Sinai experts share tips for disease prevention

Diabetes is the seventh leading cause of death in the United States. Today, 29.1 million people in the United States have been diagnosed with diabetes, according to the U.S. Centers for Disease Control and Prevention.

3 Nov 2017

Review explains nuances of non-invasive prenatal screening for pregnant women

As the use of non-invasive prenatal screening (NIPS) grows, there has been concern within the medical community that a poor understanding of this technique among clinicians and patients could negatively impact pregnancies.

3 Nov 2017

Collaborative research identifies broadly cross-protective vaccines for H3N2 influenza

A collaborative research and development partnership between researchers at the University of Georgia and Sanofi Pasteur, the largest influenza vaccine manufacturer in the world, has resulted in the identification of a vaccine candidate that protects against multiple co-circulating strains of H3N2 influenza isolated over five seasons following testing in mouse and ferret models.

2 Nov 2017

Efficacy News and Research

New gene therapy trial for X-linked myotubular myopathy offers hope to children

Major labeling inaccuracies in cannabidiol extract products

Study assesses representation of women and minorities in clinical drug trials

Mislabeling of cannabidiol products poses safety and efficacy issues for consumers

SBP researchers identify new protective function for brain protein genetically linked to Alzheimer's

FDA approves first targeted cancer treatment based on basket study

FDA grants approval to first treatment for rare blood cancer

FDA approves new drug Zelboraf for Erdheim-Chester disease

Sleepless nights lead to sluggish brain activity

Single dose of measles vaccine not sufficient to protect children against disease in Bangladesh

Trial indicates blood-plasma infusions for Alzheimer’s patients are safe and may be effective

Digital health coaching program improves well-being, decreases symptoms for RA patients

Many patients with knee, hip and spine osteoarthritis use opioids to manage chronic pain

Internet-based educational program improves health-related quality of life for teens with JIA

Low-energy ESWT shows promise for managing digital ulcers in patients with systemic sclerosis

Researchers develop new drug screening method for rapid, accurate identification of fentanyl

ACADIA Pharmaceuticals announces results from Phase II study of antipsychotic in Alzheimer's disease psychosis

Diabetes awareness month: Mount Sinai experts share tips for disease prevention

Review explains nuances of non-invasive prenatal screening for pregnant women

Collaborative research identifies broadly cross-protective vaccines for H3N2 influenza

Bringing real-time analytics to downstream bioprocess monitoring

Designing fluidic architecture for scalable diagnostic platforms

Evaluating new approaches for rare disease genomics

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