UK campaign for access to treatments and support for those with spinal muscular atrophy
Currently, there are two approved treatments for SMA: risdiplam and nusinersen. These treatments were initially approved temporarily under Managed Access Agreements while the NHS and NICE gathered data to assess their cost-effectiveness. Now, as these agreements come to an end, both treatments face a crucial review.
SMA UK are proud to join together with Treat SMA and MDUK to bring you this webinar about NICE’s upcoming Multi Technology Assessment for Spinraza and Risdiplam. 2024 sees the end of the managed access agreements (MAAs) for the two treatments. An expert committee will gather to assess the new clinical and real-world evidence, along…
This year the Managed Access Agreement for Spinraza and Risdiplam expires. The MAA was put in place five years ago so the pharmaceutical companies could gather more evidence to resubmit to NICE for approval on the NHS with Biogen extending their MAA by a year.
The Medicines and Health Products Regulatory Agency (MHRA) has given marketing authorisation to Evrysdi®▼(risdiplam) for the treatment of 5q spinal muscular atrophy (SMA) in individuals with a clinical diagnosis of SMA Type 1, Type 2 or Type 3 or with one to four SMN2 copies.
In a groundbreaking discovery, researchers have identified a previously unknown mechanism that contributes to the progressive degeneration of motor neurons in spinal muscular atrophy (SMA). This breakthrough opens up new avenues for treatment, potentially leading to more effective therapies that can halt or even reverse the disease’s course.
Today is ‘Worldwide Stop The Pressure Day’ The objective this day is to increase awareness about pressure injury prevention and to educate the public on this topic. Please see the link for more info and a short video on statistics. 2023WWPIPD – National Pressure Injury Advisory Panel (npiap.com)
Tonight Pathfinders are hosting UpLift EMPOWER Marketing with Martyn Sibley from Purple Goat Agency – an event you don’t want to miss! Limited spaces for young people with a muscle weakening condition. 📆 Tuesday 7th November 2023 ⌚ 18:00 – 19:00 📍 Online Webinar 💷 FREE 📝 Sign up – https://UpLiftEMPOWER.eventbrite.co.uk Are you aged 18-30?…
Purple Tuesday is a global social movement and the number one brand for improving the customer experience for disabled people and their families 365 days a year. This includes accessibility and in inclusivity for disabled consumers. Quoted from the ‘Purple Tuesday’ website “The initiative inspires leadership and staff of organisations from all sectors and sizes…
Truthfully, we have been gone, despite being a little quiet, we continue to fight hard behind-the-scenes for formal approval next year for all of the available treatments.
