Previous studies have shown that protein expression of DOCK7 is increased in skeletal muscle biopsies from patients with Duchenne muscular dystrophy (DMD), leading researchers from the University of Alabama at Birmingham and affiliated organizations to assess the functional impact of DOCK7 on normal muscle and embryonic development of zebrafish.

Alzamend Neuro Inc. recently presented pharmacokinetic data for the lithium-based sparing therapy AL-001, a lithium salicylate/L-proline co-crystal, in 5XFAD mice, a murine model of Alzheimer’s disease (AD).

Researchers from the University of Queensland recently provided details on the discovery and preclinical characterization of a new hematopoietic prostaglandin D2 synthase (HPGDS) inhibitor, CLS-189, being developed for the treatment of Duchenne muscular dystrophy (DMD).

The endocannabinoid system is an endogenous neuromodulatory system that is highly expressed in the central nervous system (CNS) and peripheral tissues and is known to play an important role in modulating learning and memory processes, where the cannabinoid CB1 receptor is the most widely expressed G protein-coupled receptor in the CNS, although its presence is also high in peripheral tissues.

Epicrispr Biotechnologies Inc. has secured $68 million in the first close of its series B financing to support the clinical development of EPI-321, a first-in-class epigenetic therapy for facioscapulohumeral muscular dystrophy (FSHD).

The first disease modifying therapies for Alzheimer’s may have limited utility in some senses, but they will be a force for change, providing momentum and altering the way governments as payers, and health systems as carers, think about the disease.