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Immunotherapy drug found to be effective in patients with advanced Merkel cell carcinoma

A multi-center phase II clinical trial investigating pembrolizumab as a first-line and programmed cell death-1 therapy in patients with advanced Merkel cell carcinoma reports lasting tumor control, generally manageable side effects and improved overall survival.

8 Feb 2019

New acute porphyria treatment shows promising results in clinical study

Acute porphyria is a group of uncommon diseases that can cause severe, potentially life-threatening attacks of abdominal pain, nausea, vomiting and paralysis. Liver transplantation is currently the only effective treatment available for the most seriously afflicted patients.

7 Feb 2019

Study: Promising new treatment for children with ADHD shows improvement in efficacy

A new study in children aged 6 to 12 years of dasotraline, a promising new treatment for attention deficit hyperactivity disorder, showed significant improvement in efficacy in the treatment of ADHD compared to placebo beginning at week 1 and continuing throughout the study.

4 Feb 2019

SCYNEXIS announces positive results from first interim efficacy analysis of FURI study

SCYNEXIS, Inc., a biotechnology company delivering innovative therapies for difficult-to-treat and often life-threatening infections, today announced positive results from the first interim efficacy analysis of the ongoing FURI study.

1 Feb 2019

Human trials set to begin for testing new 'fat pill' in overweight patients

Are you overweight and living in the South East of England? If so, you could help scientists who need volunteers to test a new 'fat pill'.

29 Jan 2019

NIAID-funded clinical trial of Ebola vaccines is underway at Cincinnati Children's

A Phase 1 clinical trial of investigational vaccines intended to protect against Zaire ebolavirus (Ebola) is underway at Cincinnati Children's Hospital Medical Center in the United States. The study is sponsored by the National Institute of Allergy and Infectious Diseases, part of the National Institutes of Health.

28 Jan 2019

New class of antisense oligonucleotide therapeutics tested in humans

A first-in-human study with a new class of antisense oligonucleotide therapeutics showed the ability to target the RNA-silencing drug to the liver, resulting in improved potency and safety at therapeutic doses. The design and results of this trial, conducted in healthy human volunteers are reported in Nucleic Acid Therapeutics, a peer-reviewed journal from Mary Ann Liebert, Inc. publishers.

28 Jan 2019

Investigational Ebola treatment found to be safe in adults

The investigational Ebola treatment mAb114 is safe, well-tolerated, and easy to administer, according to findings from an early-stage clinical trial published in The Lancet.

25 Jan 2019

Combination of cystic fibrosis drugs found to be safe and effective in children aged 2-5 years

An open label Phase 3 study conducted at Ann & Robert H. Lurie Children's Hospital of Chicago and other centers established that a combination of cystic fibrosis drugs lumacaftor and ivacaftor is safe and effective in children aged 2-5 years, whose disease is caused by two copies of F508del-CFTR gene mutation - the most common and severe form of cystic fibrosis.

25 Jan 2019

Clinical testing begins for temperature-stable experimental tuberculosis vaccine

Vaccinations have begun in a Phase 1 human clinical trial testing a freeze-dried, temperature-stable formulation of an experimental tuberculosis vaccine candidate.

23 Jan 2019

Experimental drug can positively modify key characteristic behavior in FXS patients

Researchers at MIND Institute at UC Davis and Rush University Medical Center have found that mavoglurant, an experimental drug known as a mGluR5 negative modulator, can positively modify a key characteristic behavior in individuals with fragile X syndrome.

22 Jan 2019

Combination therapy shows high efficacy in treating people with leishmaniasis and HIV

The results of clinical trials conducted in Ethiopia by the Drugs for Neglected Diseases initiative, in partnership with Médecins Sans Frontières, the University of Gondar, and Addis Ababa University, open the way for more effective and safer treatments for people with both HIV and visceral leishmaniasis, a group of patients who have historically suffered from poor treatment options.

18 Jan 2019

MD Anderson Cancer Center collaborates with Dragonfly for new immunotherapy drug clinical trials

The University of Texas MD Anderson Cancer Center and Dragonfly Therapeutics, Inc., today announced a strategic collaboration to bring Dragonfly's TriNKET™ immunotherapy drug candidates to patients in clinical trials beginning in 2019.

16 Jan 2019

Investigational topical cream may be effective for treating atopic dermatitis

A study published in the British Journal of Dermatology indicates that an investigational nonsteroidal topical cream may be effective for treating atopic dermatitis, one of the most common inflammatory skin diseases.

9 Jan 2019

New editorial proposes changes to improve minority representation in clinical trials

In an editorial published in CNS Spectrums, Jay Avasarala, MD, PhD, takes the research community to task for its lack of minority representation in Phase III clinical trials for drugs to treat Multiple Sclerosis (MS).

2 Jan 2019

Multicenter study demonstrates effectiveness, safety of topical antibiotics in NICU babies

A team of doctors led by Karen L. Kotloff, M.D., University of Maryland School of Medicine, Center for Vaccine Development and Global Health, has performed a clinical trial involving multiple hospitals that tested the effectiveness of applying a topical antibiotic known as mupirocin for prevention of Staphylococcus aureus infection in babies in the neonatal intensive care unit.

28 Dec 2018

New vaccine shows promise against chikungunya virus

A live vaccine genetically engineered from a common measles vaccine promises to be effective against the chikungunya virus. Such is the central finding of a recently completed Phase II trial now published in the prestigious journal "The Lancet".

21 Dec 2018

FDA supports C-Path's Type 1 Diabetes Consortium to pursue biomarker qualification

Critical Path Institute (C-Path) announced today that its Type 1 Diabetes (T1D) Consortium has received a positive response to its Letter of Intent (LOI) from the U.S. Food and Drug Administration (FDA) detailing the FDA's decision to accept the consortium's Biomarker Initiative project into the Center for Drug Evaluation and Research (CDER) Biomarker Qualification Program (BQP).

19 Dec 2018

New therapy for childhood blindness shows 'very promising' results

A new therapy aimed at improving the sight of people with one of the most common forms of childhood blindness, has shown 'very promising' initial results, according to a study involving UCL researchers.

18 Dec 2018

Intraocular injection improves vision in a form of congenital retinal blindness

A new treatment for patients with a form of congenital retinal blindness has shown success in improving vision, according to results published today in Nature Medicine led by researchers at the Scheie Eye Institute in the Perelman School of Medicine at the University of Pennsylvania.