LAS VEGAS — Muscular dystrophies encompass a range of muscle disorders resulting from genetic mutations. Over time, these conditions lead to muscle weakness, which hinders everyday activities. Muscular dystrophy takes various forms, each targeting specific muscle groups and manifesting at different ages with varying degrees of severity. It can either...
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TORRANCE, Calif. — Emmaus Life Sciences, Inc. (OTCQX: EMMA), a leader in sickle cell disease (SCD) treatment, today announced that it has received marketing authorization from the Puerto Rico Department of Health for Endari® (L-glutamine oral powder). This approval marks a significant milestone in Emmaus’ mission to improve the lives...
ROCKVILLE, Md. – Emmes, a global, full-service Clinical Research Organization (CRO) dedicated to supporting the advancement of public health and biopharmaceutical innovation, today announced a partnership with MedGenome aimed at accelerating breakthrough treatments, powered by human genomics, for rare disease patients. “This is an exciting opportunity to partner with the...
Bath, England – When you’re an autistic teenager living with chronic pain, getting treatment for your pain can be a challenging experience. That’s according to a group of young people who’ve spoken to Dr. Abbie Jordan of the Department of Psychology and Centre for Pain Research at The University of...
Adding enasidenib to azacitidine significantly improved responses in older patients with newly diagnosed, IDH2-mutant acute myeloid leukemia (AML), according to research published in The Lancet Oncology. In a phase 1b/2 trial (ClinicalTrials.gov Identifier: NCT02677922), researchers compared enasidenib plus azacitidine with azacitidine monotherapy. The trial enrolled 107 patients with newly diagnosed, IDH2-mutant AML who were...
SOUTH SAN FRANCISCO, Calif. — Encoded Therapeutics Inc., a biotechnology company focused on developing genetic medicines for severe central nervous system (CNS) disorders, today announced the approval of its Clinical Trial Authorisation (CTA) application by the United Kingdom (UK) Medicines and Healthcare products Regulatory Agency (MHRA) for the EXPEDITION Study of...
SOUTH SAN FRANCISCO, Calif. — Encoded Therapeutics Inc., a clinical-stage biotechnology company developing genetic medicines for severe neurological disorders, today announced the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to ETX101 following review of preliminary seizure data from patients treated in its ongoing...
SOUTH SAN FRANCISCO, Calif. — Encoded Therapeutics Inc., a biotechnology company focused on developing genetic medicines for severe central nervous system (CNS) disorders, today outlined the global development strategy for its lead gene therapy candidate, ETX101, for the treatment of SCN1A+ Dravet syndrome. Individuals with Dravet syndrome, the most common...
SOUTH SAN FRANCISCO, Calif. — Encoded Therapeutics Inc., a clinical-stage genetic medicines company, today announced upcoming presentations of interim clinical data from its ongoing POLARIS Phase 1/2 open-label trials evaluating ETX101 in children with SCN1A+ Dravet syndrome. The data will be featured at the American Epilepsy Society Annual Meeting, taking place...
The foundation End AxD, a nonprofit dedicated to research, treatment and a cure for Alexander disease, has donated $74,000 to UMass Chan Medical School to explore a gene therapy treatment for the fatal neurological disease. The funding will go to Jun Xie, PhD, associate professor of microbiology & physiological systems,...