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Lysogene Announces First Patient Dosed with LYS-GM101 Investigational Gene Therapy for the Treatment of GM1 Gangliosidosis


Lysogene Announces First Patient Dosed with LYS-GM101 Investigational Gene Therapy for the Treatment of GM1 Gangliosidosis
Ongoing enrollment of a total of 16 patients in the UK, US and France
Regulatory News:
Lysogene (Paris:LYS) (FR0013233475 LYS), a phase 3 gene therapy platform company targeting central nervous system (CNS) diseases, today announced dosing of the first patient, at Royal Manchester Children s Hospital, part of Manchester University NHS Foundation Trust, in the global adaptative-design clinical trial with LYS-GM101 (NCT04273269), a gene therapy for the treatment of GM1 gangliosidosis.
This trial is an interventional, multi-center, single-arm, two-stage adaptive-design study evaluating the intracisternal delivery of a recombinant adeno-associated virus vector serotype rh.10 (AAVrh.10) carrying the human ß-galactosidase gene (GBL1). The clinical trial includes a safety phase and a confirmatory efficacy phase. The trial will enroll 16 patients wi ....

United Kingdom , United States , Simon Jones , Karen Aiach , Manchester University , European Union , University Of Manchester , University Dental Hospital Of Manchester , Sarepta Therapeutics Inc , Foundation Trust , Second Lysogene , Royal Manchester Children , Founder Chairman , Chief Executive Officer , Chief Investigator , Paediatric Inherited Metabolic Disease , Senior Lecturer , Rare Pediatric Disease , Casimir Trials , Sarepta Therapeutics , Manchester Royal Infirmary , Saint Mary , Manchester Royal Eye Hospital , University Dental Hospital , Trafford General , Altrincham Hospital ,

Mighty Quinn (three) who has to wear a false leg is pride of Burnley after completing 30 mile challenge walk to raise £4,500


Submitting.
Quinn and her parents were thrilled to be met by Humphrey bear at the end of the 30 Miles My Way challenge
Her mum and dad, Natasha Thomas and James Stansfield, also surprised her with some personalised balloons.
Viv Williamson, Senior Events Manager for Royal Manchester Children’s Hospital charity said: “Quinn has been an absolute superstar throughout her fundraising. When you see her face light up with a big grin it really drives home why we exist as a charity- to put smiles on children’s faces.
When Quinn s mum spotted the challenge online she decided it would be the perfect way for her daughter to get some physiotherapy while also raising money for a fantastic cause. ....

United Kingdom , Greater Manchester , United Kingdom General , Natasha Thomas , James Stansfield , Quinn Stansfield , Manchester University Dental Hospital , Withington Community Hospital , Worsthorne Primary School , Royal Manchester Children Hospital Charity , Manchester Foundation Trust Charity , Towneley High School In Burnley , Humphrey Bear , Senior Events Manager , Royal Manchester Children , Von Willebrand , Amniotic Band Syndrome , Towneley High School , Manchester Royal Infirmary , Wythenshawe Hospital , Manchester Royal Eye Hospital , Trafford General Hospital , ஒன்றுபட்டது கிஂக்டம் , அதிகமானது மான்செஸ்டர் , ஒன்றுபட்டது கிஂக்டம் ஜநரல் , நடாஷா தாமஸ் ,

Bravery of little girl with a prosthetic leg who is walking thirty miles to say thank you to hospital


Bravery of little girl with a prosthetic leg who is walking thirty miles to say thank you to hospital
Determined Quinn Stansfield will not let her disability beat her
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Brave three-year-old Quinn Stansfield won t let the matter of having a prosthetic leg stop her when it comes to walking thirty miles to raise funds for the hospital that helped her. ....

United Kingdom , Greater Manchester , United Kingdom General , Freddie Xavi , Natasha Thomas , James Stansfield , Hughie Higginson , Quinn Stansfield , Manchester University Dental Hospital , Withington Community Hospital , Royal Manchester Children Hospital Charity , Charity Senior Events , Manchester Foundation Trust Charity , Royal Manchester Children , Saint Mary , Von Willebrand , Amniotic Band Syndrome , Manchester Royal Infirmary , Wythenshawe Hospital , Manchester Royal Eye Hospital , Trafford General Hospital , Senior Events Manager Viv Williamson , Royal Manchester Childrens Hospital , ஒன்றுபட்டது கிஂக்டம் , அதிகமானது மான்செஸ்டர் , ஒன்றுபட்டது கிஂக்டம் ஜநரல் ,