CLINICAL TRIALS, STUDIES, REGISTRIES, DATA

“RGX-202 is the only next generation gene therapy for Duchenne in a pivotal phase trial. The new data from the age 1-3 cohort builds on the favorable safety and efficacy profile seen in ages 4 and older and reinforces the potential of RGX-202 to serve a wide age range of patients,” said Steve Pakola, M.D., Chief Medical Officer of REGENXBIO.

Published online March 19 in the journal Nature, the study mapped the activity of a group of nerve cells in the bird’s brain called the central nucleus of the anterior arcopallium (AAC), which is known to strongly influence the muscles in its vocal organ. Different groups of AAC cells were found to produce sounds akin to consonants and vowels.

Vidofludimus Calcium’s Activation of Nurr1 reduces neuronal loss and injury directly and Indirectly by decreasing microglial activations in preclinical models.

The Audiogene trial is designed to evaluate the safety, tolerability and efficacy of intra-cochlear injection of SENS-501 for the treatment of OTOF gene-mediated hearing loss in paediatric patients aged between 6 and 31 months at the time of gene therapy treatment. By targeting the first years of life, when brain plasticity is at its peak, the chances of these young children with pre-linguistic hearing loss of acquiring normal speech and language are maximised. The trial is comprised of two cohorts of two doses followed by an expansion cohort at the selected dose.

This article gives an overview of the new clinical trial regulations, explaining how key changes will impact clinical research, and how this new accelerated access to clinical innovation will ultimately impact patient care.

“Neuropathy is incredibly common among the aging population, regardless of disease states. There is currently no known cure for neuropathy, and most treatments address acute symptoms with no lasting impact,” says Dr. Ramona von Leden, VP of Research and Clinical Affairs for NeuFit. “NeuFit’s specialized approach to rehab focuses on supporting the function of the nervous system, and we are seeing remarkable results on patients with neuropathy.”