A Phase I/II clinical trial of the investigational gene therapy SGT-001 designed to treat Duchenne muscular dystrophy has had its clinical hold lifted by the US Food and Drug Administration…
Prana Biotechnology has announced that it has begun recruiting healthy volunteers for its Phase I clinical trial of the experimental drug PBT434. The drug is thought to affect processes linked…
According to a story from IRB Barcelona, a recent conference has brought together researchers from around the world that are dedicated to the study of the cell cycle and two…
According to Financial Buzz Chiasma, Inc. has reached a new clinical stage in its development of a treatment for acromegaly. The trial, known as CHIASMA OPTIMAL, tests a treatment currently…
Data from a Phase II clinical trial of the experimental treatment reldesemtiv for the treatment of spinal muscular atrophy has been announced. The drug has shown positive effects in the…
According to a story from globenewswire.com, the corporation Retrotope recently announced the initiation of single-patient, expanded access trials of its experimental product RT001. The patients involved have familial encephalopathy with neuroserpin…
According to Pulmonary Fibrosis News, a study with mice showed that a certain compound found in plants, parthenolide, is associated with a sizable reduction in lung fibrosis and the subsequent inflammatory…
An experimental gene therapy designed to treat Duchenne muscular dystrophy has produced exciting preliminary results in a Phase 1/2a clinical trial. Sarepta Therapeutics, the company behind the therapy’s development, announced…
An open-label extension of a Phase II study has shown that the experimental drug lenabasum is fairly safe and tolerable in patients who have diffuse cutaneous systemic sclerosis. The full…
According to a story from Newswise, a team of scientists and researchers have recently completed research that managed to reveal the genetic nature of the unusual rare disease neuromyelitis optica.…
According to a story from UK Healthcare, a team of researchers and scientists at UK Healthcare is preparing to start a clinical trial that will test a new therapy aimed…
The experimental gene therapy LentiGlobin for the treatment of severe sickle cell disease is being investigated in an ongoing Phase 1 clinical trial. Bluebird Bio, the company developing the therapy,…
Clinical data from a Phase 2 study of the experimental drug X4P-001-RD for the treatment of WHIM syndrome has been announced at the European Hematology Association’s 23rdCongress in Stockholm. The…
According to a story from BioPortfolio, the corporation Herantis Pharma Plc recently announced that its Phase 2 clinical trial measuring the effectiveness of Lymfactin, a gene therapy, in the treating…
According to a story from globenewswire.com, the biopharmaceutical company Idera Pharmaceuticals recently released the data from its Phase 2 trial for IMO-8400, an investigational product in development for the treatment…
According to Bright Surf, a recent study has led researchers to believe that children with neuroblastoma are at a higher risk of developing long-term psychological impairment. Neuroblastoma is a rare…
The first patient has undergone stem cell transplant treatment in the third cohort of a Phase I clinical study of the experimental treatment ISC-hpNSC for Parkinson’s disease. The full article can…
The Live Like Bella Childhood Cancer Foundation was created for Bella Rodriguez-Torres. The Miami born girl had stage four of a rare childhood cancer which took her life at the…
The first patients have been enrolled in two clinical trials of the experimental drug neridronate. The trials are part of the company’s Phase 3 program to evaluate the effects of…
Researchers have discovered a new drug that appears to slow cell growth in mantle cell lymphoma. The full article can be read here, at the Bellvitge Biomedical Research Institute (IDIBELL)…
The All of Us program is a huge research project set up by the National Institute of Health (NIH) that aims to collect genetic and health data from a representative…
Based on reports from PRNewswire, there appears to be a rising concern in both the number of cases and the amount of global concern about NASH. NASH stands for Nonalcoholic…
A phase 1/2 study of the drug lumasiran for the treatment of primary hyperoxaluria Type 1 has produced encouraging results, reports Alnylam Pharmaceuticals. The full article can be read here,…
According to a story from coloradocancerblogs.org, a recent study demonstrated that data gathered from PET scans could help providers tailor their treatment more precisely in cases of esophageal cancer, resulting…
Promising data from the ongoing Phase I clinical trial Explorer has been announced by Blueprint Medicines Corporation. The trial is designed to evaluate the effects of the experimental drug avapritinib…
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