Campaign for Access to Treatments for Spinal Muscular Atrophy

TreatSMA are extremely happy to announce that Lianne Harris has joined the team as a volunteer to assist with Fundraising and Events Co-ordination.

We are now listed as a charity on Amazon Smile. By choosing us as your selected charity Amazon will donate 0.5% of the net purchase price (excluding VAT, returns and shipping fees) of eligible purchases to us. You can set…

Charitable.radio is an amazing station focused on raising awareness of charities from all walks of life. You can listen on the Internet or using your  smart device by simply asking it to play charitable radio.

Novartis has made the decision to discontinue development of branaplam, an investigational oral, once-weekly RNA splicing modulator, for the treatment of spinal muscular atrophy (SMA). This was a difficult decision that was made as the result of rapid advancements in the SMA treatment landscape in recent years.

We received news yesterday that 241 people are now enrolled on the EAMS for Risdiplam.

We don’t normally do blog posts of this nature, but we felt that this particular piece of assistive technology is so good that we wanted to share it with the community.

A petition has been set up to get Newborn Screening funded immediately for Spinal Muscular Atrophy.