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Study examines effect of herbal medicine product on bedwetting in children

A new clinical trial investigating whether an herbal medicine product can benefit school-age children experiencing bedwetting is being led by a collaboration between researchers from the Australian Research Centre in Complementary and Integrative Medicine at the University of Technology Sydney and the Office of Research at Endeavour College of Natural Health.

5 Mar 2019

Experimental treatment offers hope to restore cells damaged in Parkinson's disease

A pioneering clinical trials program that delivered an experimental treatment directly to the brain offers hope that it may be possible to restore the cells damaged in Parkinson's disease.

27 Feb 2019

Pioneering trial offers hope for patients with Parkinson’s disease

In a pioneering new clinical trial, researchers have succeeded in delivering a cell-restoring drug directly to the brains of patients with Parkinson’s disease.

27 Feb 2019

New analysis highlights increasing focus of early-stage clinical trials on rare cancers

The proportion of early-stage drug trials tackling the biggest cancer killers has declined sharply since the early 1990s as less common tumor types receive increasing attention, according to new research to be presented during the International Congress on Targeted Anticancer Therapies 2019, taking place from today to 27 February in Paris, France.

25 Feb 2019

Clinical study shows tolerability and safety of investigational drug in boys with DMD

Patients with Duchenne muscular dystrophy have few treatment options. Medications currently available or in development either target only a subset of DMD patients with a particular genetic mutation or cause significant side effects.

23 Feb 2019

Phase 1 data reinforce safety profile of new drug for treating Duchenne muscular dystrophy

Patients with Duchenne muscular dystrophy (DMD) have few treatment options. Medications currently available or in development either target only a subset of DMD patients with a particular genetic mutation or cause significant side effects.

21 Feb 2019

Drug increases melanin production in some people with albinism

A small pilot clinical study at the National Eye Institute suggests that the drug nitisinone increases melanin production in some people with oculocutaneous albinism type 1B, a rare genetic disease that causes pale skin and hair and poor vision.

21 Feb 2019

'Trojan horse' drug shows promise for untreatable cancers

A new cancer drug that uses a ‘Trojan horse’ approach to destroy cancer cells from within has shown promising results for patients with untreatable cancers.

8 Feb 2019

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