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22nd August 2024
Our Research Summary for August 2024 is ready to view. Our Research Summary includes information about recent leukodystrophy research and clinical trials, including article summaries and direct links to websites and articles.
22nd August 2024
National Tay-Sachs & Allied Diseases Association Publishes GM2 Voice of the Patient Report based on NTSAD hosted, Externally-Led Patient-Focused Drug Development (EL-PFDD) Meeting for GM2 with members of the FDA.
20th August 2024
Adrenal insufficiency survey – project, led by Edge Hill University is working with NHS clinicians to develop national education standards and resources for sick day/emergency management of adrenal insufficiency in children and young people.
7th August 2024
A recent research publication suggests the estimated prevalence of adult-onset leukoencephalopathy with axonal-spheroids and pigmented glia (ALSP) is underreported in the U.S., EU, and UK.
2nd August 2024
Passage Bio shared they entered into a new agreement with GEMMA Biotherapeutics for the out-licensing of three paediatric gene therapy programs. Read their full update.
31st July 2024
Our Research Summary for July 2024 is ready to view. It includes information about recent leukodystrophy research and clinical trials, including article summaries and direct links to websites and articles.
31st July 2024
The July edition of our monthly news round-up has been sent out today. It includes charity news, our community and ways to get involved.
26th July 2024
My Neuro Survey is to help better understand support individuals affected by neurological conditions. It’s completely anonymous and takes around 20 minutes to complete.
25th July 2024
How does your rare disease impact your everyday life? Complete Rare Barometer survey and share your experiences. The survey is open to people living with a rare disease and their family members from any country in the world
23rd July 2024
Important retrospective cohort study characterising mortality and the burden of illness in men with AMN in the US. Abstract and article are available to read.
16th July 2024
Mirum Pharmaceutical submits new drug application to FDA for Chenodiol for the treatment of CTX which if approved, means chenodiol would be the first and only medication approved to treat patients with CTX.
8th July 2024
In the latest Alexander Disease podcast, Dr. Amy Waldman and Dr. Natasha Snider discussed several recent AxD research publications. The articles are looking at anesthesia and AxD, and GFAP cross-linking to itself.
