Alex The Leukodystrophy Charity (Alex TLC)

Michael's story was featured in the Paisley Daily Express 📰 This is important in raising awareness of ALD 💙 Our CEO, Sara Hunt, was featured in the article providing a comment about screening for ALD and our efforts to get ALD added to the newborn screening programme in the UK. To find out more about our 'Screen Our Boys' campaign: https://lnkd.in/e3i2VPeW To read the article: https://lnkd.in/gcf-VRyy #ALD #alextlc #screenourboys

Promising update from Mirum Pharmaceuticals, Inc. “Following the landmark RESTORE data, we are excited about the potential to have an approved treatment option that may reduce the progressive symptoms associated with this rare disease,” said Chris Peetz, chief executive officer at Mirum. “If approved, chenodiol would be the first and only medication approved to treat patients with CTX, enabling earlier diagnosis and treatment of these life-altering symptoms.” Full press release: https://lnkd.in/entXR35a #CTX #leukodystrophy

A fantastic job opportunity with Genetic Alliance UK 📑✍ They are looking for a new Senior Policy and Research Officer to join their team Questions? Email recruitment@geneticalliance.org.uk Closing Date is 28th July To find out more and to apply: https://lnkd.in/efknR4HV

Not only is September Leukodystrophy Awareness Month, it’s also our 20th birthday! Blue is the official leukodystrophy awareness colour, so we’d love you to join in the celebrations by holding a ‘Wear it Blue Day’ at your school or workplace. It’s simple, those wishing to take part don their blue get up (silliness encouraged) and donate what they can to Alex TLC. You can also post your blue looks on social media to help spread vital awareness. Find out more: https://lnkd.in/eXYFM2NW #alextlc #helptocope #helptohope #wearitblue #leukodystrophy

We need your help! Unfortunately, we’ve now filled all available places within our current 2024 Community Weekend budget. Please donate to our appeal if you can, and help more people to attend and form vital connections this October, thank you. https://lnkd.in/exG-J_aR #alextlc #helptocope #helptohope #leukodystrophy #raredisease #donation #donate #charity #help #nonprofit #love #support #community #fundraising #donations #giveback #givingback #makeadifference #helpingothers

“Building on foundational work to establish a method for identifying MLD through newborn screening, there has been tremendous progress over the past few years to advance screening efforts around the world,” said Leslie Meltzer, Ph.D., chief medical officer of Orchard Therapeutics. “As with many rare, life-threatening diseases, early detection and diagnosis is key to ensuring the best possible outcomes for patients. We recognize the valuable contributions of researchers, physicians, patient advocates and families to help ensure the pace of newborn screening coincides with biomedical innovation, and we look forward to continuing to support the efforts of the broader community to accelerate MLD NBS globally.” Read full press release: https://lnkd.in/dAkzGJ8a #MLD #leukodystrophy

What could you do with an extra £87+ next month? The current prize pot for our August 50/50 Cash Draw is £86.50, and it increases with every new entry! You can enter the monthly draw for a whole year for just £12. That's just £1 per month! Or you can pay a monthly subscription of £2 for 2 entries per month. Sign up now: https://lnkd.in/e8rADHMU #alextlc #cashdraw #charitydraw #cashprize #win

Myrtelle has announced that their gene therapy, rAAV-Olig001-ASPA for the treatment of Canavan disease has been chosen for the Support for Clinical Trials Advancing Rare Disease Therapeutics (START) Pilot Program “Acceptance into the START pilot program is an honor in that it recognizes rAAV-Olig001-ASPA as a candidate for accelerated development as a potential treatment for Canavan disease. Opening the lines of communications beyond traditional meeting pathways provides the opportunity to quickly address development issues that would otherwise delay progression to market application", said Nancy Barone Kribbs, PhD, Senior Vice President of Global Regulatory Affairs at Myrtelle. To read press release: https://lnkd.in/ghKXZTeX #canavandisease #leukodystrophy

Alexander Disease Podcast 🔊 Dr. Amy Waldman and Dr. Natasha Snider discussed several recent AxD research publications - including anesthesia and AxD, and GFAP cross-linking to itself To listen: https://lnkd.in/esSK6qn6 #alexanderdisease #leukodystrophy

Cure EB’s Butterfly Run 🦋 is a celebration of inclusivity taking place at the Queen Elizabeth Olympic Park in London on Sunday 29th September 2024. They are encouraging people of all abilities to complete a 1k, 5k or 10k race. If you'd like to take part and raise funds for Alex TLC, just visit: https://lnkd.in/eV7FGnDA #alextlc #helptocope #helptohope #leukodystrophy #cureb #butterflyrun