An experimental drug for a rare, inherited form of amyotrophic lateral sclerosis (ALS) has shown promise in a phase 1/phase 2 clinical trial conducted at Washington University School of Medicine in St. Louis, Massachusetts General Hospital in Boston and other sites around the world and sponsored by the pharmaceutical company Biogen Inc. The trial indicated […]
Experimental drug shows early promise against inherited form of ALS, trial indicates
An experimental drug for a rare, inherited form of amyotrophic lateral sclerosis (ALS) has shown promise in a phase 1/phase 2 clinical trial conducted at Washington University School of Medicine in St. Louis, Massachusetts General Hospital in Boston and other sites around the world and sponsored by the pharmaceutical company Biogen Inc. The trial indicated […]
Gene therapy to boost fetal hemoglobin continues to do well in sickle cell trial
A pilot gene therapy treatment for sickle cell disease, restoring patients’ ability to make fetal hemoglobin, has produced good results in the first three patients to receive it. Investigators at Boston Children’s Hospital reported the findings of their ongoing clinical trial this week at the American Society of Hematology (ASH) annual meeting. The three adult […]
Drug trial seeking first ever treatment for dangerous side effect of Prader-Willi syndrome
One in every 15,000 children is affected by Prader-Willi syndrome (PWS), a complex, genetic endocrine condition caused by a disorder of chromosome 15. It’s non-inheritable, meaning the condition isn’t passed down from a family member. Among the regular side effects like growth hormone deficiency, hypothyroidism, delayed puberty, behavioral problems and learning difficulties, but hyperphagia is […]
Unique cancer drug discovered with help from Advanced Light Source begins historical clinical trial
An investigational cancer drug that targets tumors caused by mutations in the KRAS gene will be evaluated in phase 2 clinical trials, following promising safety and efficacy results in preliminary human studies and excellent results in animal studies. The drug, developed by Amgen and currently referred to as AMG 510, is the first therapy to […]
‘Spin’ found in over half of clinical trial abstracts published in top psychiatry journals
‘Spin’—exaggerating the clinical significance of a particular treatment without the statistics to back it up—is apparent in more than half of clinical trial abstracts published in top psychology and psychiatry journals, finds a review of relevant research in BMJ Evidence Based Medicine. The findings raise concerns about the potential impact this might be having on […]
First randomised trial finds no substantial difference in risk of acquiring HIV for 3 different forms of contraception
Previous research has suggested that some contraceptive methods may increase women’s susceptibility to HIV. However, all three forms of contraception trialled were safe and highly effective, supporting their continued and increased access alongside high quality HIV prevention services. A randomised trial of more than 7,800 African women found that a type of contraceptive injection (intramuscular […]
Gene therapy for gamma-sarcoglycanopathy moves toward a clinical trial
Isabelle Richard’s team, a CNRS researcher in an Inserm unit at Genethon, the AFM-Telethon laboratory, has demonstrated the efficacy of gene therapy and determined the effective dose for treating a rare muscle disease, gamma-sarcoglycanopathy, in mouse models of the disease. Based on these encouraging results, published in Molecular Therapy: Methods and Clinical Development, the researchers […]
Getting teens to follow strict diets in the Fast Track trial is risky, but so is obesity itself
The Fast Track to Health study is a year-long dietary trial in adolescents with obesity. Since it started in Sydney and Melbourne at the end of 2018, it has been criticised for increasing the risk of eating disorders in people who may be especially vulnerable to these conditions. Sydney-based clinical psychologist Louise Adams started up […]
Favorable outcomes seen in long term for ALLR3 trial
(HealthDay)—For children with B-cell precursor acute lymphoblastic leukemia with late bone marrow relapse, risk stratification by minimal residual disease seems to be an effective strategy for treatment, according to a study recently published in The Lancet Haematology. Catriona Parker, Ph.D., from the University of Manchester in the United Kingdom, and colleagues conducted a long-term follow-up […]