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UPDATE ON UK APPROVAL OF GENE THERAPY - Click here to view this entry

UPDATE ON UK APPROVAL OF GENE THERAPY

Sat Jul 10 2021
NICE unfortunately leans towards a ‘NO’ for the treatment of Metachromatic Leukodystrophy (MLD).
NICE (National Institute of Clinical Excellence) the drug decision making body have publicly released their interim decision today (9 July 2021) not to recommend OTL-200 (Libmeldy), the treatment for individuals suffering from both, late infantile and early juvenile Metachromatic Leuko....
NBS Campaign Update - Click here to view this entry

NBS Campaign Update

Tue Jun 8 2021
Further to our recent meeting with Baroness Blackwood, the newborn screening stakeholdler group has been working on reviewing our campaign. Given the huge number of health issues now jostling for priority as we are beginning see some light at the end of the Covid tunnel, we have decided to streamline our campaign into 3 clear key policy changes which would address the problems with the s....
A lovely surprise  - Click here to view this entry

A lovely surprise

Fri May 21 2021
Ava was blown away by a surprise parcel all the way from America today, containing a beautiful blanket handmade by her dear friend Ella. Ella (and her brother Eli) received gene therapy in Milan just before Ava and our families have become firm friends. Ella is currently dedicating all of her spare time to making these unique gifts for friends and people in her community, always thinking of others....
RIP Grandad George  - Click here to view this entry

RIP Grandad George

Mon Apr 19 2021
Today we laid to rest my (Georgina) Dad, Ava's Grandad George, on what would have been his 80th birthday. Covid has complicated procedures and limited our options for his funeral, but nevertheless we gave him a send-off that we hope he would be proud of. I met thousands of stars in my life as a television producuer before we had ever heard of MLD, but none of them ever shone as brightly as he....
NBS Campaign Update - Click here to view this entry

NBS Campaign Update

Fri Apr 16 2021
Today Nickie Aiken MP and I met with Baroness Nicola Blackwood (Chairperson) and Dr. Richard Scott (Clinical Lead for Rare Disease)  from Genomics England, in order to discuss next steps our newborn screening campaign and how we can align with Genomics England in their important work in this area.

The UK has made incredible strides in genomic diagnositics and there is much enthusi....
Happy 7th Re-Birthday - Click here to view this entry

Happy 7th Re-Birthday

Sun Mar 28 2021
Another small celebration with just the 4 of us, but a no less importnat one. Today is the 7th anniversary of Ava receiving her new DNA and the day she was given a fighting chance against the monster that is MLD. We are forever grateful to the heroic scientists and medics who have made this possible and for the truly delightful girl we have the pleasure of every single day. 
Sad News - Click here to view this entry

Sad News

Mon Mar 22 2021
Beyond sad to report that Ava's Grandfather George has passed away. George suffered from chronic arthritis from his early 20's and a lifetime of medication finally took a toll on his heart and kidneys at the age of 79. Despite this, he was a constant beacon of optimism, positivity, wisdom and humour in our lives, enduring 10 major joint replacements without complaint and never allowing h....
Rare Disease Day 2021 - Click here to view this entry

Rare Disease Day 2021

Sun Feb 28 2021
Today is Rare Disease Day when we are thinking of all of the courageous and inspirational families who are living with rare diseases like MLD. At times it's hard to believe the surreal year which has passed since last year's RDD, when we launched the ArchAngel Newborn Screening Review Campaign in conjunction with Nickie Aiken MP.  Whilst Covid has understandably knocked us off ....
Happy New Year - Click here to view this entry

Happy New Year

Sun Jan 24 2021
We have had a quiet and subdued Christmas and been pretty sad not to see our closest family members. However Covid is still clearly in charge and we are doing our best to protect Ava as someone clincially vulnerable and also to adhere to the neccessary guidlines for the safety of everyone else. We hope that the new year brings more positivity and good health to all. In the meantime, the snow has g....
Update on the UK approval of gene therapy for MLD - Click here to view this entry

Update on the UK approval of gene therapy for MLD

Fri Dec 18 2020
ArchAngel MLD Trust and Ava's mother Georgina were nominated as 'expert witnesses' and both have just submitted extensive evidence to The National Institute of Health & Care Excellence (NICE) in support of an application to have Gene Therapy for MLD approved for use by NHS England. If approved, this remarkable treatment could offer UK children with a timely diagnosis the same se....

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