Neurology

Roche paused shipments of the Duchenne muscular dystrophy gene therapy in countries that reference US FDA approval of Elevidys after the agency asked Sarepta to pause US shipments; it remains available in Japan and Brazil.

The company had planned to keep its DMD gene therapy available for ambulatory patients, but now says pausing shipments may enable a better working relationship with the US FDA while safety labeling is updated.

The biotech company is planning for Phase III based on positive topline results from Vibrance-1 in narcolepsy type 1 and remains optimistic about type 2 disease.

Sarepta knew about the death in a Phase I trial of SRP-9004 for LGMD when it recently revealed a restructuring plan, but did not disclose the event. The now-discontinued gene therapy candidate uses the same AAV vector as Elevidys, which has been linked to two DMD patient deaths.

In this week's Digital Health Roundup, Medtech Insight's team discusses progress on autonomous surgery, Medtronic's new partnership with IRCAD, FDA cybersecurity news, and C-suite interviews with OpenWater, Flow Neuroscience and EnsoData.

The drugmaker announced positive results from two Phase III trials for the drug in narcolepsy type 1 that analysts said bode well for the OX2R agonist class as a whole.

The updated US FDA label modifies dose titration based on TRAILBLAZER-ALZ 6, a study that showed a more gradual rise in dosing levels reduced ARIA-E incidence without reducing amyloid clearance.

The US Federal Circuit’s March 2025 decision affirming a lower court’s determination on both induced infringement and non-obviousness concerning a key patent shielding Janssen’s Invega Trinza (paliperidone palmitate) long-acting injectable is set to stand – for now.

A discussion with Karen Harris, CFO of the Alzheimer's Drug Discovery Foundation, about the foundation's investment strategy, biotech and investor sentiment at the recent BIO conference and what innovations give her hope for Alzheimer's patients.

Eisai and Lilly explain why they disagree with today’s conclusion by the health technology assessment institute, NICE, that the benefits of their respective drugs for Alzheimer’s disease are too small to justify the additional cost to the National Health Service.

Lerodalcibep and palbociclib are among the latest new drugs that the European Medicines Agency has started to review for potential EU marketing authorization.

Devika Wood, CEO of Brain+, explains the importance of developing health tech solutions for dementia and the growing need to both raise awareness and improve overall access to nondrug interventions like CST.