10:06 Overview of therapeutic approaches for CTNNB1 Syndrome - Dr. Ana Gonzalez Hernandez CTNNB1 Foundation 83 views - 8 months ago
12:54 Manufacturing for clinical readiness: GMP and quality control - Cristina Martin Quintin (Viralgen) CTNNB1 Foundation 43 views - 8 months ago
41:20 GAIN trial: A phase I/II open-label clinical trial in patients with CTNNB1 Syndrome CTNNB1 Foundation 270 views - 8 months ago
18:04 Navigating regulatory complexities: Health authority submissions - Dr. Elise Destree CTNNB1 Foundation 22 views - 8 months ago
16:33 Cross-border access to paediatric clinical trials in Europe: language inclusion CTNNB1 Foundation 12 views - 8 months ago
11:20 Selecting the optimal route of gene therapy delivery - Dr. Peter Spazzapan, PhD (UMC Ljubljana) CTNNB1 Foundation 68 views - 8 months ago
19:17 Understanding safety and toxicology in small and large animals - Dr. Duško Lainšček CTNNB1 Foundation 32 views - 8 months ago
19:44 What do pre-clinical models tell us about vector distribution? - Dr. Ruud Bueters CTNNB1 Foundation 29 views - 8 months ago
13:15 Challenges and experiences of families living with CTNNB1 syndrome - Tina Nikolić CTNNB1 Foundation 81 views - 8 months ago
16:24 Assessing AAV-mediated gene therapy in preclinical models of CTNNB1 syndrome - Dr. Andrea Iturralde CTNNB1 Foundation 52 views - 8 months ago
20:31 Gene therapy today: turning science into cures - Dr. Leszek Lisowski CTNNB1 Foundation 155 views - 8 months ago
14:28 Consequences of pathogenic mutations on b-catenin conformational properties - Dr. Sonia Bañuelos CTNNB1 Foundation 32 views - 8 months ago
20:49 Genotype-phenotype characterization and functional reconstitution of pathological β-catenin variants CTNNB1 Foundation 85 views - 8 months ago
14:37 Clinical, psychological and neuropsychologicalcharacterisation of 25 CTNNB1 syndrome patients CTNNB1 Foundation 42 views - 8 months ago
15:30 Genotype-phenotype correlations in CTNNB1 neurodevelopmental syndrome - Dr. Nina Žakelj CTNNB1 Foundation 92 views - 8 months ago
23:56 From fear to a new life: Our experience with brain gene therapy for AADC deficiency CTNNB1 Foundation 101 views - 8 months ago
45:42 CTNNB1 Foundation: Our dreams become reality – counting months to a hope! - Dr. Špela Miroševič CTNNB1 Foundation 93 views - 8 months ago
12:33 3rd International CTNNB1 Syndrome Conference - Opening Ceremony CTNNB1 Foundation 108 views - 8 months ago
17:50 CRISPR-based Prime edit strategy for correction of CTNNB1 mutations - Dr. Vida Forstnerič CTNNB1 Foundation 40 views - 8 months ago
17:50 Update on small molecules developed by the Italian Institute of Technology - Dr. Angelo Serani CTNNB1 Foundation 35 views - 8 months ago
19:34 Small molecule efficacious therapeutic treatment for CTNNB1 syndrome - Dr. Michele Jacob CTNNB1 Foundation 367 views - 8 months ago
1:53 Help us deliver hope: The final push for CTNNB1 gene therapy CTNNB1 Foundation 632 views - 8 months ago
2:14 SYSNAV Healthcare supports CTNNB1 Foundation: Revolutionizing patient care with Syde technology CTNNB1 Foundation 353 views - 1 year ago
13:56 2nd International CTNNB1 Syndrome Conference - Opening Cerimony CTNNB1 Foundation 152 views - 1 year ago
10:05 Accelerating Patient Access to Biomedical Advancements in Slovenia - Dr. Mojca Benčina CTNNB1 Foundation 91 views - 1 year ago
22:12 CTNNB1 Foundation: A Path to Hope for Children with CTNNB1 Syndrome - Dr. Spela Mirosevic CTNNB1 Foundation 428 views - 1 year ago
13:09 Introduction to Non-clinical Safety Testing for CTNNB1 Gene Therapy - R. Bueters CTNNB1 Foundation 49 views - 1 year ago
19:24 AAV-mediated gene therapy for CTNNB1 Syndrome - Dr. Leszek Lisowski and Dr. Andrea Perez Iturralde CTNNB1 Foundation 377 views - 1 year ago
20:10 Small Molecule Efficacious Therapeutic Treatment for CTNNB1 Syndrome - Dr. Michele Jacob CTNNB1 Foundation 94 views - 1 year ago
17:43 Presenting 80 UK Patients with Pathogenic CTNNB1 Variants - Taisiia Sazonova CTNNB1 Foundation 68 views - 1 year ago
14:46 Conference Conclusion & Gratitude with Marta Santiago, Dr. Damjan Osredkar and Dr Špela Miroševič CTNNB1 Foundation 120 views - 1 year ago
14:24 Clinically Relevant Endpoints: Continuous Movement Measure in Real Life - Dr. Laurent Servais CTNNB1 Foundation 67 views - 1 year ago
18:25 From Diagnosis to Starting a Gene Therapy Clinical Trial for children with AGU, Julia Taravella CTNNB1 Foundation 104 views - 1 year ago
21:39 The Journey of CTNNB1 PAOs Leading Therapy Development - Emily Amerson and Dr Ana Gonzalez Hernandez CTNNB1 Foundation 39 views - 1 year ago
17:39 Overview of Routes of Administration in Gene Therapy with a Focus on ICV - Thomas Carrol CTNNB1 Foundation 195 views - 1 year ago
25:50 Co-creating Hope in the Community of Children and Families Affected by CTNNB1 - Dr David Gosar CTNNB1 Foundation 42 views - 1 year ago
20:14 Breaking Barriers: Promoting Communication and Managing Behaviours - Megan Ryder Stanley CTNNB1 Foundation 55 views - 1 year ago
27:41 Congenital Heart Defects in CTNNB1 Syndrome: Raising Clinical Awareness - Dr. Lorenzo Sinibaldi CTNNB1 Foundation 117 views - 1 year ago
16:43 CTNNB1-Related Movement Disorders: A Complex But Recognizable Movement Disorders Unit - Bambino Gesù CTNNB1 Foundation 128 views - 1 year ago
14:11 Challenges of transitioning to adolescent and adulthood - Dr. Laura de Graaff CTNNB1 Foundation 41 views - 1 year ago
13:34 Understanding Epilepsy Cases in CTNNB1 Syndrome: Insights and Implications - Shivang Khandelwal CTNNB1 Foundation 74 views - 1 year ago
16:00 Understanding the Cellular Basis of Corpus Callosum Crossing Defect in CTNNB1 - Dr Arpan Parichha CTNNB1 Foundation 136 views - 1 year ago
17:47 The “Dragonfly Study”: CTNNB1 Syndrome International Natural History Study - Dr. Damjan Osredkar CTNNB1 Foundation 92 views - 1 year ago
23:17 Fostering Effective Communication Skills in Your Child – Neurobiological Therapy - Magda Malinowska CTNNB1 Foundation 22 views - 1 year ago
12:01 Safety Measures in Preclinical Development: Example from CTNNB1 Study - Dr. Jan Prochazka CTNNB1 Foundation 85 views - 1 year ago
26:30 Meeting the Challenge of Rare Disease in the Family: 25 Years of Lessons Learned - Dr Al Freedman CTNNB1 Foundation 43 views - 1 year ago
15:06 What our Genotype-Phenotype Study Taught us About Rare Symptoms in CTNNB1 - Dr. Nina Žakelj CTNNB1 Foundation 359 views - 1 year ago
26:17 RNA-centred Strategies for Upregulation of β-catenin in CTNNB1 - Dr Petra Sušjan and Matea Maruna CTNNB1 Foundation 56 views - 1 year ago
34:28 Genetics of CTNNB1 in the Context of Other Neurodevelopmental Disorders - Stephan J. Sanders CTNNB1 Foundation 126 views - 1 year ago
18:19 The Value of Mindfulness Practice for Patients with Rare Disease - Dr Špela Miroševič CTNNB1 Foundation 15 views - 1 year ago
13:03 Tethered Cord with Surgical Detethering, an Under-Recognised Comorbidity - Dr. Sandrine Cornaz Buros CTNNB1 Foundation 461 views - 1 year ago
13:24 Evaluation of the Efficacy of CTNNB1 AAV9 Therapy on a Mouse Model - Dr. Duško Lainšček CTNNB1 Foundation 141 views - 1 year ago
17:01 Managing Expectations with Gene Therapy - Dr. Laurent Servais CTNNB1 Foundation 153 views - 1 year ago
14:23 The Manufacture of a Hope: Projected Timelines - Cristina Martín, Project Manager Viralgen CTNNB1 Foundation 50 views - 1 year ago