fashion -WSS

fashion

author：leisure time 2025-09-30 17:30:04 - browse：88447

A vial of Tofersen injection on the left of its box — The ALS drug tofersen is marketed as Qalsody in the U.S. Biogen

LONDON — Neurologists and patient advocates are up in arms over a policy decision by a U.K. health agency that they say will imperil access to an ALS treatment that’s available in the U.S. and on its way to approval in the European Union.

The medicine in question, Biogen’s tofersen, is designed to treat a rare genetic form of the neurodegenerative disease, accounting for some 2% of all ALS cases. And the issue is how the U.K.’s cost-effectiveness agency has said it would assess the costs and benefits of the therapy.

The agency, the National Institute for Health and Care Excellence, or NICE, has a special pathway for drugs for very rare diseases, which advocates and doctors argue is the route through which tofersen should be reviewed. But NICE has indicated it would review the drug through its standard process, saying it’s because ALS as a whole does not count as a “very rare” disease. That sets a higher bar to clear.

Get unlimited access to award-winning journalism and exclusive events.

Subscribe Log In

Previous article： Guidelines to prevent youth baseball injuries need more muscle
Next article： Neuroscience’s long legacy of racism

WSS

Readout LOUD podcast: FTC v. biotech, future of Pfizer and Vertex

Health AI adoption puts security, privacy startups on investors' radar

Virtual Event: March of the Biosimilars

New Medicare drug pricing powers could soon be upended in court

As JPM 2024 winds down, some health tech firms project optimism

Risky weight loss products often used by teens globally, per study

focus

fashion

knowledge

Apple is now the first public company to be valued at $3 trillion

Virtual Event: March of the Biosimilars

At least 13 dead in Texas as scorching temps continue

Nurses recount patients attacks

FDA approval of Sarepta Duchenne genetic therapy gives me hope

Physicians and social media: If you see something, say something?

Apple is now the first public company to be valued at $3 trillion

Methadone treatment changes are coming. Do they go far enough?

Supreme Court strikes down use of affirmative action

Physicians and social media: If you see something, say something?

leisure time

Readout LOUD podcast: 2023 in review, and a look at biotech in 2024

Three issues to watch in global health in 2024

Caregivers of transplant patients call for more support, training

Fauci’s Covid grilling leads GOP to weigh foreign research rules

Argenx readies a blockbuster opportunity with drug for autoimmune disease

Readout Newsletter: Geron, Novo Nordisk, WuXi updates

Wikipedia

focus

hotspot

Wikipedia