01:57

Our 20th Birthday Message from Karen and Sara

09:41

The Royal Society of Medicine presentation

12:01

Community Member Presentation

35:41

Disability Rights UK Presentation

02:34

Community Weekend Video

14:44

Alex TLC Update - Sara Hunt

20:32

Bladder Care - Dr Sarah Wright

18:15

Queen Square Adult Leukodystrophy Group - Dr David Lynch

21:40

Mental Health for Patients, Carriers and Carers - Dr Pujit Gandhi

31:03

Minoryx Therapeutics SL Clinical Programme Update - Dr Arun Mistry, Minoryx Therapeutics

19:14

Genetics & Genetic Counselling - Dr James Poulter

24:27

ALD Monitoring, Personal Story - Kathleen O'Sullivan Fortin

24:41

VK0214 in Patients with AMN, Update - Dr Joel Neutel, VikingTherapeutics

09:07

SwanBio Program Update, investigating AAV gene therapy for AMN - Eileen Sawyer, SwanBio Therapeutics

21:31

Paediatric Symptom Management - Alasdair Parker

19:36

Gene Therapy for Adrenomyeloneuropathy AMN - Dr Florian Eichler

21:24

Newborn Screening Study - Jennifer Jones

24:23

Sex-specific newborn screening for X-linked Adrenoleukodystrophy (ALD) - Dr Stephan Kemp

21:47

Summary of Saturday's Research Roundtable - Dr James Poulter

06:49

Gene Therapy For Canavan Disease - Dr Florian Eichler, SwanBio Therapeutics

16:53

Aicardi Goutières Syndrome - Dr Yanick Crow, University of Edinburgh MRC Human Genetics Unit

16:41

Era of Gene Therapy, Modifying the Neurological Outcomes of MLD - Dr Dipak Ram, Orchard Therapeutics

12:55

Adult-Onset Leukoencephalopathy, Anoxal Spheroids & Pigmented Glia - Dr Charles Wade, Vigil Neuro

24:28

FBX-101 Clinical Program for Patients with Krabbe Disease - Juan Ruiz, Forge Biologics

19:13

Quality of life and lifestyle intervention for women with ALD - Wolfgang Kohler

21:26

Symptoms and Treatments in Women with Adrenoleukodystrophy (ALD) - Dr Florian Eichler

14:24

Experience of being a young carrier - Taylor Kane

18:50

Working with Alex TLC - NHSE IWMD Service & Registry - Helen Morris & Toni Mees

20:44

Transplant for Advanced Cerebral ALD, Risk-Benefit - Dr Troy Lund

23:44

The psychosocial impact on parents having a child diagnosed with cerebral ALD - Dr Hilary Piercy

21:06

Support for severely symptomatic patients adults - Dr Charles Wade & Prof. Jeremy Chattaway

19:26

NHS England Inherited White Matter Disorder (IWMD) Service and Patient Registry - Dr Rahul Singh

20:16

Leukolabs, a UK Research Collaboration - Dr Noémie Hamilton

20:27

Hematopoietic Stem-Cell Transplant (HSCT), Adults with Adult Onset Cerebral ALD - Dr Wolfgang Kohler

18:15

ALD, Monitoring children - Dr James Davison

18:52

Adrenomyeloneuropathy AMN, Symptoms and treatments for men - Dr Robin Lachman

21:13

Gene Therapy:Bone Marrow Transplant - Dr Claire Horgan

26:20

Expectations of patient care and impact of new NHS England Inherited White Matter Disorder Service –

24:19

New Born Screening for Adrenoleukodystrophy Round Table Summary – Prof Colin Steward

30:14

Metachromatic Leukodystrophy MLD Update – Prof Nicole Wolf

21:59

Leriglitazone research update Adrenoleukodystrophy and Adrenomyeloneuropathy – Minoryx Therapeutics

17:03

Natural History Study & Patient Reported Outcome Measures (Adrenomyeloneuropathy) - SwanBio

12:11

Long term impacts of Bone Marrow Transplant Q&A with Karen Harrison and Claire Turner

13:06

Bone Marrow Transplants and Gene Therapies - Orchard: Katie Snell

11:32

ON373 Research Update (Alexander Disease) - Dr Amy Waldman

27:56

Managing Mental Health – Dr Pujit Gandhi

12:14

ON373 Research Update (Alexander Disease) -  Katrina Moore PhD

06:44

Alex TLC Update 2021 - with Sara Hunt and Karen Harrison

28:51

Support for Severely Symptomatic Patients (adults) - Dr David Lynch & Prof. Jeremy Chataway

33:34

Genetic Counselling and Informing Wider Family - Jon Wells

28:59

Support for Severely Symptomatic Patients (children) -  Dr Alasdair Parker

14:16

Long term impacts of Bone Marrow Transplant - Claire Turner

34:34

Managing Continence Symptoms - Dr Sarah Wright

35:22

Transition to adult services - Cambridge Rare Disease Network

28:46

NHS England Inherited White Matter Disorders Patient Registry - Dr John Livingstone

15:31

Gene Therapy UpdateGM1 Gangliosidosis & Krabbe - PassageBio

28:16

PXL770 and PXL065 for Adrenomyeloneuropathy – AMN Research Update - Poxel

05:11

In Memory - for those we have lost to Leukodystrophy

01:22

Our Big Give Christmas Challenge Campaign 2021

01:22

Children's support tools for a Rare Disease - Big Give Christmas Challenge 2021