European Prize for Women Innovators: Špela Miroševič
1:30
European Prize for Women Innovators: Špela Miroševič
2 ай бұрын
Mednarodna konferenca sindroma CTNNB1, junij 2024
3:02
Mednarodna konferenca sindroma CTNNB1, junij 2024
2 ай бұрын
2nd International CTNNB1 Syndrome Conference - Opening Cerimony
13:56
2nd International CTNNB1 Syndrome Conference - Opening Cerimony
4 ай бұрын
The Discovery of CTNNB1 Syndrome - Dr. Joep De Ligt
4:59
The Discovery of CTNNB1 Syndrome - Dr. Joep De Ligt
4 ай бұрын
Accelerating Patient Access to Biomedical Advancements in Slovenia - Dr. Mojca Benčina
10:05
Accelerating Patient Access to Biomedical Advancements in Slovenia - Dr. Mojca Benčina
4 ай бұрын
CTNNB1 Foundation: A Path to Hope for Children with CTNNB1 Syndrome - Dr. Spela Mirosevic
22:12
CTNNB1 Foundation: A Path to Hope for Children with CTNNB1 Syndrome - Dr. Spela Mirosevic
4 ай бұрын
Introduction to Non-clinical Safety Testing for CTNNB1 Gene Therapy - R. Bueters
13:09
Introduction to Non-clinical Safety Testing for CTNNB1 Gene Therapy - R. Bueters
4 ай бұрын
AAV-mediated gene therapy for CTNNB1 Syndrome - Dr. Leszek Lisowski and Dr. Andrea Perez Iturralde
19:24
AAV-mediated gene therapy for CTNNB1 Syndrome - Dr. Leszek Lisowski and Dr. Andrea Perez Iturralde
4 ай бұрын
Small Molecule Efficacious Therapeutic Treatment for CTNNB1 Syndrome - Dr. Michele Jacob
20:10
Small Molecule Efficacious Therapeutic Treatment for CTNNB1 Syndrome - Dr. Michele Jacob
4 ай бұрын
Presenting 80 UK Patients with Pathogenic CTNNB1 Variants - Taisiia Sazonova
17:43
Presenting 80 UK Patients with Pathogenic CTNNB1 Variants - Taisiia Sazonova
4 ай бұрын
Conference Conclusion & Gratitude with Marta Santiago, Dr. Damjan Osredkar and Dr Špela Miroševič
14:46
Conference Conclusion & Gratitude with Marta Santiago, Dr. Damjan Osredkar and Dr Špela Miroševič
4 ай бұрын
Clinically Relevant Endpoints: Continuous Movement Measure in Real Life - Dr. Laurent Servais
14:24
Clinically Relevant Endpoints: Continuous Movement Measure in Real Life - Dr. Laurent Servais
4 ай бұрын
From Diagnosis to Starting a Gene Therapy Clinical Trial for children with AGU, Julia Taravella
18:25
From Diagnosis to Starting a Gene Therapy Clinical Trial for children with AGU, Julia Taravella
4 ай бұрын
The Journey of CTNNB1 PAOs Leading Therapy Development - Emily Amerson and Dr Ana Gonzalez Hernandez
21:39
The Journey of CTNNB1 PAOs Leading Therapy Development - Emily Amerson and Dr Ana Gonzalez Hernandez
4 ай бұрын
Overview of Routes of Administration in Gene Therapy with a Focus on ICV - Thomas Carrol
17:39
Overview of Routes of Administration in Gene Therapy with a Focus on ICV - Thomas Carrol
4 ай бұрын
Co-creating Hope in the Community of Children and Families Affected by CTNNB1 - Dr David Gosar
25:50
Co-creating Hope in the Community of Children and Families Affected by CTNNB1 - Dr David Gosar
4 ай бұрын
Breaking Barriers: Promoting Communication and Managing Behaviours - Megan Ryder Stanley
20:14
Breaking Barriers: Promoting Communication and Managing Behaviours - Megan Ryder Stanley
4 ай бұрын
Congenital Heart Defects in CTNNB1 Syndrome: Raising Clinical Awareness - Dr. Lorenzo Sinibaldi
27:41
Congenital Heart Defects in CTNNB1 Syndrome: Raising Clinical Awareness - Dr. Lorenzo Sinibaldi
4 ай бұрын
CTNNB1-Related Movement Disorders: A Complex But Recognizable Movement Disorders Unit - Bambino Gesù
16:43
CTNNB1-Related Movement Disorders: A Complex But Recognizable Movement Disorders Unit - Bambino Gesù
4 ай бұрын
Challenges of transitioning to adolescent and adulthood - Dr. Laura de Graaff
14:11
Challenges of transitioning to adolescent and adulthood - Dr. Laura de Graaff
4 ай бұрын
Understanding Epilepsy Cases in CTNNB1 Syndrome: Insights and Implications - Shivang Khandelwal
13:34
Understanding Epilepsy Cases in CTNNB1 Syndrome: Insights and Implications - Shivang Khandelwal
4 ай бұрын
Understanding the Cellular Basis of Corpus Callosum Crossing Defect in CTNNB1 - Dr Arpan Parichha
16:00
Understanding the Cellular Basis of Corpus Callosum Crossing Defect in CTNNB1 - Dr Arpan Parichha
4 ай бұрын
The “Dragonfly Study”: CTNNB1 Syndrome International Natural History Study - Dr. Damjan Osredkar
17:47
The “Dragonfly Study”: CTNNB1 Syndrome International Natural History Study - Dr. Damjan Osredkar
4 ай бұрын
Fostering Effective Communication Skills in Your Child - Neurobiological Therapy - Magda Malinowska
23:17
Fostering Effective Communication Skills in Your Child - Neurobiological Therapy - Magda Malinowska
4 ай бұрын
Safety Measures in Preclinical Development: Example from CTNNB1 Study - Dr. Jan Prochazka
12:01
Safety Measures in Preclinical Development: Example from CTNNB1 Study - Dr. Jan Prochazka
4 ай бұрын
Meeting the Challenge of Rare Disease in the Family: 25 Years of Lessons Learned - Dr Al Freedman
26:30
Meeting the Challenge of Rare Disease in the Family: 25 Years of Lessons Learned - Dr Al Freedman
4 ай бұрын
What our Genotype-Phenotype Study Taught us About Rare Symptoms in CTNNB1 - Dr. Nina Žakelj
15:06
What our Genotype-Phenotype Study Taught us About Rare Symptoms in CTNNB1 - Dr. Nina Žakelj
4 ай бұрын
RNA-centred Strategies for Upregulation of β-catenin in CTNNB1 - Dr Petra Sušjan and Matea Maruna
26:17
RNA-centred Strategies for Upregulation of β-catenin in CTNNB1 - Dr Petra Sušjan and Matea Maruna
4 ай бұрын
Genetics of CTNNB1 in the Context of Other Neurodevelopmental Disorders - Stephan J. Sanders
34:28
Genetics of CTNNB1 in the Context of Other Neurodevelopmental Disorders - Stephan J. Sanders
4 ай бұрын
Пікірлер
@chrisinla24
@chrisinla24 8 ай бұрын
Where can I find the spanish version?
@chrisinla24
@chrisinla24 8 ай бұрын
THE "LIKE" button doesn't work. Thank you so much for your words. You are so right!
@chrisinla24
@chrisinla24 8 ай бұрын
👍🏽
@jesunkarim4086
@jesunkarim4086 3 жыл бұрын
Plz.. Sir All update News and Vdieo give Your Chelan Hemophilia (A) and Gene therapy😥🙂😥 20+ thousand hemophilia present in Bangladesh😥 🙂😥 I am a Hemophilia (A) present😭 And Why we globally in THIS Proper treatment?
@sarleen9916
@sarleen9916 3 жыл бұрын
The reason hemophilia A treatment is very similar problem.. And cost of treatment it can be high every medical recruitment it was high cost.. So many medical board can consider researchers can't take serious indulge this scenario... Every seconds around the world 30+thousands child born in hemophilic.. Now a days medical treatment enhance in this world's but don't consider people's.. Because less of awareness.. Everything in this world circulation is money minded.. So far now a days also same protocol we are all fall.. So main object.. Why people can't understand our feeling. Government can't consider main reasons research and medical board ready to forward use for hemophilia patient cost... Buying medical treatment and reaserch.. Some countries give me a proper. Treatment in heamophilic patients.. Some other countries can't consider.. But it's OK.. These all God hand not we are all hand.. Best of luck to all
@nejcmihelicTOPG
@nejcmihelicTOPG 3 жыл бұрын
Zelo lep ter ganljiv filmček 😉. Upam, da boste uspešni, predvsem pa da tudi vsem (pre)ostalim otrokom ne bo treba več trpeti zaradi te “bolezni” oziroma bolezni 😎. Srečno še naprej tako pri zbiranju denarja kot seveda tudi pri zdravljenju 🌞 🍀 ☘️ 😉 @CTNNB1 Foundation.
@sarleen9916
@sarleen9916 3 жыл бұрын
Hai sir i am heamophilia a patient is there any option i use this gene therapy life time cure treatment is there avail..
@jesunkarim4086
@jesunkarim4086 3 жыл бұрын
Best of luck brother 🌺🙂 Same i hemophilia present (A)
@marjakocbek5454
@marjakocbek5454 3 жыл бұрын
Samo in Špela, zelo mi je žal, da je na vaši poti težka preizkušnja. Držim pesti, da terapija uspe. Občudujem vašo voljo in pogum, da prosite za pomoč. Video je čudovit in ganljiv. Srečno, Urban.
@ctnnb1foundation238
@ctnnb1foundation238 3 жыл бұрын
Draga Marja! Najlepša hvala za sporočilo. Držite pesti za nas!
@marjakocbek5454
@marjakocbek5454 3 жыл бұрын
Držim.