Roche's Risdiplam Granted PRIME Designation by EMA
17 December 2018 / Posted in: Treatments & Research
Roche have issued a media release confirming PRIME designation has been granted by the European Medicines Agency (EMA) for risdiplam (RG7916) for the treatment of SMA.
Response from Biogen to Charities' Letter re Bridging Solution
14 December 2018 / Posted in: Treatments & Research
We have received a written response from Biogen to the joint charities' letter we sent on 29th November about the urgent need to set up a bridging solution for access to nusinersen.
Response from NICE to Charities' Two Joint Letters
14 December 2018 / Posted in: Treatments & Research
NICE have sent a letter back in response to the joint charities' two letters we sent: firstly about the urgent need to set up a bridging solution for access to nusinersen; secondly urging them to ensure nusinersen has priority place at the next meeting.
Call for Expressions of Interest: Patient Representatives wanted to participate in PARADIGM Project
13 December 2018 / Posted in: Treatments & Research
PARADIGM's objective is to develop processes and tools to guarantee patient engagement in three key decision-making points in medicines research and development. Closing date for applications is 28th December.
Biogen Issues Global Community Statement on Spinraza
13 December 2018 / Posted in: Treatments & Research
Biogen has provided a global community statement on Spinraza. The statement covers worldwide statistics, a summary of data as presented at the 23rd International Congress of the World Muscle Society, and plans to "broaden their development portfolio".
AveXis Issues U.S. Community Statement on BLA Acceptance
12 December 2018 / Posted in: Treatments & Research
AveXis has provided the following U.S. community statement on the BLA submission and acceptance for their investigational therapy, AVXS-101 - now known as ZOLGENSMA.
We are Supporting Genetic Alliance UK's ‘Resetting the Model’ Project
11 December 2018 / Posted in: Treatments & Research
We are taking every opportunity to attend APPGs to give voice to the SMA Community’s frustrations and distress caused by the NICE appraisal process for nusinersen. We are supporting this ambitious project aimed at improving the way that future decisions on rare disease medicines are made as a positive way forward.
AVXS-101 to receive expedited regulatory review in the US and Europe
06 December 2018 / Posted in: Treatments & Research
Novartis has announced that the US Food and Drug Administration (FDA) has accepted for review with Priority Status their new drug application for the experimental gene therapy for SMA, AVXS-101, which has now been renamed ZOLGENSMA.
UK SMA Research Day 2019
06 December 2018 / Posted in: Treatments & Research
The UK SMA Research Consortium and SMA UK are inviting all SMA Researchers to the next UK SMA Research Day, which will be held January 23rd 2019, from 9am to 5pm, in the Keele Hall Salvin Room at Keele University.
Biogen awarded the 2018 International Prix Galien as Best Biotechnology Product for Spinraza (nusinersen)
06 December 2018 / Posted in: Treatments & Research
While we still await a decision from NICE, this marks the seventh Prix Galien for Spinraza, following country recognitions in the U.S., Germany, Italy, Belgium-Luxembourg, the Netherlands and the U.K.
