The Annual Congress of the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) meeting in Copenhagen this week is celebrating its 40th edition. In recognition of this landmark, the plenary session and opening lecture were attended by Queen Margrethe of Denmark. Afterward, the hot topic session on neuroprotective therapies set the stage for the subsequent discussions on the latest trends in the management and treatment of multiple sclerosis (MS).

Papillon Therapeutics Inc.’s PPL-001 has been awarded orphan drug designation by the FDA for Friedreich’s ataxia. PPL-001 is an experimental gene-corrected CD34+ hematopoietic stem and progenitor cell (HSPC) therapy.

Somite Therapeutics Inc.’s lead program, SMT-M01, has been awarded orphan drug and rare pediatric disease designations by the FDA for the treatment of Duchenne muscular dystrophy (DMD). The program leverages the company’s proprietary Alphastem artificial intelligence (AI) platform to develop a novel cell replacement therapy for DMD.

The map of the human brain is not a static image of the cellular architecture of the central nervous system (CNS). Throughout life, all living beings are born, develop and age. The brain of pregnant mothers reflects a dynamic that modifies this general picture. A collaboration of scientists from the University of California has studied the changes in the adult brain during pregnancy and observed that a large part of it adapts during the development of the fetus.

Voyager Therapeutics Inc. has announced the selection of a development candidate in a gene therapy program for the treatment of an undisclosed neurological disease under its collaboration with Neurocrine Biosciences Inc.

The European Commission has granted EU orphan drug designation to Synerkine Pharma BV’s SK-01 to treat complex regional pain syndrome.

By looking at the electrical activity of tumor cells, rather than the neurons that innervate them, investigators at Baylor College of Medicine have added both basic and translational insights to the emerging field of cancer neuroscience. In their studies, which were published in Cancer Cell on Sept. 5, 2024, the researchers identified the cell of origin for IDH-mutated gliomas.

Silo Pharma Inc. has completed a pre-IND meeting with the FDA regarding the company’s development plan for SPC-15, an intranasal prophylactic treatment for post-traumatic stress disorder (PTSD) and stress-induced anxiety disorder.

Stealth Biotherapeutics Inc. has been awarded the Kyle Bryant Translational Research Award from the Friedreich’s Ataxia Research Alliance (FARA) to evaluate mitochondria-targeted molecule SBT-589 in Friedreich’s ataxia (FA).