Approve life-saving treatment for Tora

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Tora, our 6 months old daughter, was diagnosed with Spinal Muscular Atrophy (Type 1) last week.  SMA is a life-threatening neuromuscular disease that is the number 1 genetic cause of infant mortality.

She can not move her legs much anymore.  Soon she will stop moving her arms and then her neck.  And before she could celebrate her second birthday, it’s likely that she’ll stop breathing.

Tora would want to live.  She would want to travel the world, swim in the blue sea, maybe breathe in the fresh mountain air.  She would like to walk our dog in the park.

There are two potential treatments that could help Tora and other children suffering from SMA:

1. Spinraza from Biogen (  It is an approved treatment available in several countries in Europe, and Scotland.

2. AVXS-101 from AveXis, Novartis (  It is in clinical trial phase globally and approval is expected in late 2019.

Due to the high cost of such treatments (Spinraza is $750,000 in first year, $375,000 in subsequent years), most families with SMA children can not afford to keep them alive without government support.

We request National Institute for Health and Care Excellence (NICE) to approve funding for SMA treatments through NHS England.  And we also request that governments globally will support research and development of treatments for rare diseases and make such treatments available to those in need.

#LetToraLive, #LetToraBreathe