This is about our Granddaughter – it could so easily be your Granddaughter, Grandson, Daughter, Son, Niece, Nephew, etc. etc.
Please do not be as complacent as we were until a week ago.
Just read a little and try to help us help Kaysey and many others.
It seems that we are all used to receiving communications asking for help on a regular basis these days, but the following is only a request for a few minutes of your time that could make a difference to thousands of children and families in the UK.
A little over a week ago, we knew very little about SMA (Spinal Muscular Atrophy). In the last seven days since our granddaughter Kaysey, was diagnosed with SMA type 2 and after surfacing from a fog of disbelief, we have undertaken a crash course acquiring a basic knowledge and understanding of this disease.
All we are asking is for as many people as possible to read the next paragraph and to click on the link afterwards (this is a link to a Charity based website but does not involve any money/donations), but just needs a few moments of your time. Below this, is also some information on the basics of SMA.
We would urge everyone to add their voice to the possible licensing of SPINRAZA in the UK by writing to their MP and asking them to contact the EMA about approving SPINRAZA as quickly and broadly as possible. The below link also provides template letters to send and links to locate your relevant MP.
It’s easy to help:
- Locate MP through link above
- Download relevant letter for your area
- Fill out letter ready to send to your MP
- After you have completed letter and have saved, click back on to MP website and click email address
- Mail editor on your PC will open, simply attach letter and type a few words on email
- Click send and that’s it
Thank you so much for your help.
Information about SMA: In summary, Spinal Muscular Atrophy is a rare, genetically inherited neuromuscular condition.
SMA may affect crawling and walking ability, arm, hand, head and neck movement, breathing and swallowing.
SMA is often grouped into ‘Types’.
Types of SMA are based on the age at which symptoms first appear and what physical ‘milestones’ a baby or child is likely to achieve.
What can be done: SPINRAZA is the first and (at the moment) only approved treatment (generic name – nusinersen) for SMA.
In summary, SPINRAZA tinkers with defective genes. Children born with SMA do not produce enough of a protein called SMN, which is key to motor development. SPINRAZA works by amplifying the gene responsible for SMN, thereby boosting its production. SPINRAZA was approved by the FDA in America on the 23rd December 2016 for all types of SMA.
This product is being fast tracked through the European Medicines Agency approval process. We do not know exactly when a decision will be made and there is no way of knowing yet if the drug will be approved in the UK and if it is, if it will be approved for all types of SMA.
An Expanded Access Programme and clinical trials are already underway in the UK for babies with SMA type 1 but the best chance that those affected have, is for SPINRAZA to come to market as quickly as is safely possible.
The SMA Trust and Muscular Dystrophy UK are keeping people up-to-date through their websites and social media.
The latest update went out a few days ago and you can read this at the following link:
Despite everything, Kaysey is a very affectionate, happy and beautiful 14 month old little girl.
It is important to remember that SMA can affect anyone.
For Kaysey and all children like her, we owe it to them to try to make a difference.
We believe that difference lies in bringing a medicine to treat SMA to market as quickly as possible.
In the last week we have arrived at the conclusion that the answers to SMA are an issue of timing rather than funding.
For the Kaysey’s of today and tomorrow, the more quickly we can bring a suitable medicine to market, the better the chances of improving the lives of the thousands of people who are and continue to be affected by SMA.
Gary and Jenny Copp
(Proud Grandparents to Kaysey)