4th August 2026
Rare Patient Passport Video
In this 10-minute interview from EURORDIS-Rare Diseases Europe, Sarah Baker, CEO of CamRARE, explains how the Rare Patient Passport came about and the difference it can make for patients and families.
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4th August 2026
In this 10-minute interview from EURORDIS-Rare Diseases Europe, Sarah Baker, CEO of CamRARE, explains how the Rare Patient Passport came about and the difference it can make for patients and families.
30th July 2026
We’ve been selected to take part alongside 2 other charities this August.
The charity with the highest number of votes will win the donation.
28th July 2026
Alex TLC recently submitted evidence for the Scottish Medicine Consortium review of the medicine Libmeldy for approval of it through NHS Scotland for patients with metachromatic leukodystrophy (MLD).
27th July 2026
European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended granting marketing authorisation approval under exceptional circumstances for NEZGLYAL® (leriglitazone) as a treatment for male cALD patients
27th July 2026
We have been fighting to add adrenoleukodystrophy (ALD) to the UK Newborn Screening Blood Spot Programme since 2017 and submitted extensive additional evidence to the National Screening Committee (NSC) this month.
24th July 2026
Alex TLC has been working closely with Genetic Alliance as part of their Future for Rare campaign to inform what future UK rare disease policy should look like.
20th July 2026
Rare Summit 2026 is going to be a hybrid event – giving you the flexibility to join us from around the world.
20th July 2026
We’re pleased to share Genetic Alliance UK’s research report, Independent Evaluation of the Generation Study: Views of support organisations.
17th July 2026
The APBD Research Foundation is conducting a survey of patients and caregivers to prepare their community for future clinical trials – referred to as “clinical trial readiness.”
6th July 2026
For the first time, the lived experience of Alexander Disease has been documented and formally entered into the record drug developers, regulators, and researchers will reference for years.
30th June 2026
We have sent out the June edition of our monthly news round-up. Please sign up to receive correspondence from us including our news round-up.
