Today, 28th February marks #RareDiseaseDay.
February 28, 2023 9:43 amDuchenne Muscular Dystrophy is a rare disease. A condition that affects 1 in every 3,500 boys born. Alex was diagnosed with this condition shortly before his 4th birthday....
Categories for Research News
Study paves the way for improved detection of heart disease in female carriers of DMD
September 7, 2022 1:56 pmA recent report from a study funded by partly by Alex’s Wish, in collaboration with Duchenne UK has improved understanding of early detection of heart muscle disease in female carriers of Duchenne muscular dystrophy (DMD), paving the way for further research. ...
Italfarmaco announces positive topline data from Phase 3 trial showing positive benefits of Givinostat in Duchenne
July 4, 2022 1:10 pmItalfarmaco Group Announces Positive Topline Data from Phase 3 Trial Showing Beneficial Effect of Givinostat in Patients with Duchenne Muscular Dystrophy — Study meets primary endpoint with secondary and exploratory endpoints showing consistency with primary endpoint — — Data reinforces previously observed safety profile for Givinostat; treatment in boys with DMD continues to show a good tolerability profile — — The company is planning to discuss the potential for marketing application submission with regulatory authorities for Givinostat in DMD — — Results were presented at the Annual PPMD Conference on June 25, 2022 — MILAN, Italy, June 25, 2022 – Italfarmaco Group announced today positive topline data from its completed Phase 3 EPIDYS trial with Givinostat, the company’s proprietary histone deacetylase (HDAC) inhibitor, in boys with Duchenne Muscular Dystrophy (DMD)....
Important Update on Alex’s Clinical Trial
June 30, 2022 11:49 amThis is great news for the Duchenne community. We would like to share this news with all of our wonderful supporters....
Positive Update from Vamorolone Trial
June 7, 2021 12:47 pmSteriods are the main stay treatment for those living with Duchenne, Alex has been on them since he was 4, they come with lots of nasty side effects, including poor bone health – Alex broke his hip when he was just 9 years old.As...
#CTD2021
May 20, 2021 9:34 amToday is Clinical Trials Day, the work we do at Alex’s Wish is to fund clinical trials that will hopefully one day see Duchenne cured for good! ...
Alex’s Wish help fund study to improve our knowledge of heart care in patients with DMD
May 12, 2021 9:52 am12 May 2021 Alex’s Wish has invested £10,000 towards a study funded by Duchenne UK that aims to improve our knowledge of treating heart muscle disease in patients with Duchenne muscular dystrophy (DMD). ...
Alex meets Marianela Schiava
January 20, 2021 2:38 pmThe last time that Alex visited Newcastle he was so pleased to be able to meet Marianela Schiava. Marianela is the Clinical Research Associate, a position that Alex’s Wish funded £30,000 towards, alongside Duchenne UK....
Gene Therapy Update
January 13, 2021 10:53 amThis is exciting news for all affected by Duchenne. Gene therapy cannot come soon enough it’s one of the things we are all working so hard to get....
Thank you and Merry Christmas
December 17, 2020 1:39 pmAs we near the end of 2020, we’ve been reflecting on what’s been achieved together. We started the year with huge plans, with the momentum that we had established we were sure that this would be our most successful year yet! ...
Update on Vamorolone Study
October 7, 2019 9:11 amTop Line Data of 18-month Vamorolone treatment of Duchenne Muscular Dystrophy patients shows continued improvement of symptoms with reduction in Corticosteroid safety concerns....
Solid Bioscience Gene Therapy Update
August 19, 2019 8:49 amAlex’s Wish have received a positive update from Solid Bioscience on their gene therapy trial called IGNITE DMD which Alex’s Wish invested in....
End of Year Update from Emma
December 20, 2018 9:07 pmAs 2018 comes to end, I wanted to share with you an update on how this year has gone. For a start, it’s been our best year in terms of fundraising....
Update on Gene Therapy
July 3, 2018 12:53 pmIn recent years, our community has watched with hope as pre-clinical data has emerged showing the promise of gene therapy as a valuable treatment for everyone living with Duchenne Muscular Dystrophy. ...
Alex’s Wish gives £50,000 grant to Solid-GT
October 15, 2016 7:53 amAlex’s Wish partners with Solid-GT and funds important Gene Therapy Development We would like to thank all of our supporters who have enabled us to give a £50,000.00 grant to Solid-GT to support their Gene Therapy Development....
Translarna first ever drug will treat 50 boys in England
April 20, 2016 1:43 pmWe were thrilled to hear the news last Friday, that NICE have given the green light on Translarna – the first ever drug to treat the underlying cause of Duchenne for those eligible. ...
First genetic cure for rare diseases!
April 28, 2015 11:41 amNew era of medicine begins as first children cured of genetic disorder The pioneering therapy offers hope to the hundreds thousands of people suffering from inherited conditions This story gives hope to boys living with Duchenne Muscular Dystrophy....