Evelina London Children's Hospital

Stallergenes Greer Announces Publication of Positive Results for PALFORZIA® Phase 3 Study in Peanut-allergic Children Aged 1 to 3 Years

Retrieved on: 
Monday, November 20, 2023
Research, Fitness & Nutrition, Clinical Trials, Other Health, Health, Pharmaceutical, General Health, Other Science, Science, Safety, Prevalence, Hope, Patient, Death, NHS foundation trust, Pediatric Allergy and Immunology, Peanut allergy, AIT, Geoffrey Guy, Evelina London Children's Hospital, Hospital, Pharmaceutical industry, Poseidon

Stallergenes Greer, a leading global healthcare company specialising in allergen immunotherapy (AIT), announces the publication of results from the Phase 3 POSEIDON (Peanut Oral Immunotherapy Study of Early Intervention for Desensitization) study in the New England Journal of Medicine Evidence.

Key Points: 
  • Stallergenes Greer, a leading global healthcare company specialising in allergen immunotherapy (AIT), announces the publication of results from the Phase 3 POSEIDON (Peanut Oral Immunotherapy Study of Early Intervention for Desensitization) study in the New England Journal of Medicine Evidence.
  • The study evaluated the efficacy and safety of PALFORZIA [defatted powder of Arachis hypogaea L., semen (peanuts)] in peanut-allergic children aged 1 to 3 years.
  • Overall, 84.7% of patients on PALFORZIA and 93.8% of patients on placebo completed the study.
  • The results, published in the New England Journal of Medicine Evidence, are truly promising and potentially life-changing for young patients.

BioMarin to Present Pipeline at Upcoming R&D Day on Tuesday, September 12th

Retrieved on: 
Wednesday, September 6, 2023
Medical genetics, FRCP, University, Royal Free Hospital, University of the Witwatersrand, BioMarin Pharmaceutical, Consultant, Keck School of Medicine of USC, Achondroplasia, Children's Hospital, Guy's and St Thomas' NHS Foundation Trust, National Health Laboratory Service, Charlotte Maxeke Johannesburg Academic Hospital, University of Southern California, BMRN, Division, University of Minnesota Medical School, SAN, NHS foundation trust, Evelina London Children's Hospital, Hospital, Haemophilia, Master of Medicine, Hemostasis, Broadcasting, Instrumentation, Pediatrics, Bachelor of Medicine, Bachelor of Surgery, Endocrinology

SAN RAFAEL, Calif., Sept. 6, 2023 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN), a global biotechnology company dedicated to transforming lives through genetic discovery, will host an R&D Day at 8:00 a.m. ET on Tuesday, September 12, 2023. BioMarin management and external experts will provide an update to the investment community on the company's development portfolio.

Key Points: 
  • R&D Day to be Webcast Beginning at 8:00 a.m. Eastern Time September 12th
    SAN RAFAEL, Calif., Sept. 6, 2023 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN), a global biotechnology company dedicated to transforming lives through genetic discovery, will host an R&D Day at 8:00 a.m.
  • ET on Tuesday, September 12, 2023.
  • BioMarin management and external experts will provide an update to the investment community on the company's development portfolio.
  • An archived version of the remarks will also be available through the Company's website for a limited time following the event.

BridgeBio Pharma Announces Positive Feedback from the U.S. FDA and EU EMA on the Regulatory Path for a Pivotal Phase 3 Trial of Infigratinib in Children with Achondroplasia

Retrieved on: 
Wednesday, September 6, 2023
ENDO, Osteochondrodysplasia, FGFR3, Evelina London Children's Hospital, ASHG, Marketing, Pharming, PALO, Aes, Achondroplasia, Șaeș, EMA, Physician, Cancer, European, European Medicines Agency, Growth, FDA, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, News, Food, Hypochondroplasia, Safety, Vaccine, Pharmaceutical industry, EU, AHV

”The regulatory feedback clearing the path to approval for infigratinib is great news for children living with achondroplasia and for physicians who treat them.

Key Points: 
  • ”The regulatory feedback clearing the path to approval for infigratinib is great news for children living with achondroplasia and for physicians who treat them.
  • ”We are thrilled to have regulatory alignment on key elements of our Phase 3 trial design and the path towards submitting a marketing application to both the FDA and the EMA.
  • Additionally, BridgeBio expects to initiate clinical development for infigratinib in hypochondroplasia, a skeletal dysplasia closely related to achondroplasia and similarly driven by FGFR3 gain-of-function variants.
  • BridgeBio has previously presented promising preclinical data for hypochondroplasia at ENDO 2023 and ASHG 2022.

BridgeBio Pharma to Host Educational Roundtable with Esteemed Achondroplasia Expert on February 16, 2023

Retrieved on: 
Monday, February 13, 2023
Achondroplasia, Cancer, NHS foundation trust, Hospital, Webcast, Guy, Evelina London Children's Hospital, PALO, Osteochondrodysplasia, Guy's and St Thomas' NHS Foundation Trust, Nursing

Dr. Irving, a key opinion leader who specializes in genetic and rare conditions and specifically in skeletal dysplasia, will provide an overview of the medical complications as well as the social and emotional impact experienced by those living with achondroplasia.

Key Points: 
  • Dr. Irving, a key opinion leader who specializes in genetic and rare conditions and specifically in skeletal dysplasia, will provide an overview of the medical complications as well as the social and emotional impact experienced by those living with achondroplasia.
  • Dr. Irving has established multidisciplinary pediatric clinics to help those with the condition, and is an active researcher, hoping to develop new treatment paradigms and potentially improve the clinical outcome of severe genetic skeletal conditions.
  • To access the live webcast, please visit the “Events” page within the Investors section of the BridgeBio website at http://investor.bridgebio.com .
  • A replay of the webcast will be available on the BridgeBio website for 90 days following the event.

Aimmune Therapeutics to Present Positive Results from Phase 3 POSEIDON Study of PALFORZIA® [Peanut (Arachis hypogaea) Allergen Powder-dnfp] in Peanut-Allergic Children Aged 1 to 3 Years

Retrieved on: 
Thursday, November 10, 2022
Biotechnology, Pharmaceutical, Health, Clinical Trials, ACAAI, Review, AES, Pediatric Allergy and Immunology, Guy's and St Thomas' NHS Foundation Trust, Evelina London Children's Hospital, Parent, Diagnosis, NHS foundation trust, University of Arkansas, Therapy, Food, Hospital, News, Guy Goodwin, Safety, Medicine, OIT, Anaphylaxis, University, Patient, Peanut allergy, Death, Ageing, Medical Affairs Bureau, FDA, Peanut, American College, Pharmaceutical industry, Copper, Health insurance, Allergy, Pediatrics

The trial evaluated the efficacy and safety of PALFORZIA [Peanut (Arachis hypogaea) Allergen Powder-dnfp] in peanut-allergic children aged 1 to 3 years.

Key Points: 
  • The trial evaluated the efficacy and safety of PALFORZIA [Peanut (Arachis hypogaea) Allergen Powder-dnfp] in peanut-allergic children aged 1 to 3 years.
  • The encouraging results of the POSEIDON study indicate that PALFORZIA has the potential to be an effective and convenient treatment option for peanut-allergic children aged 1 to 3 years.
  • POSEIDON enrolled 146 patients, with 98 randomized to PALFORZIA and 48 randomized to placebo.
  • Overall, 84.7% of patients on PALFORZIA and 93.8% of patients on placebo completed the study.

Venus Medtech Obtains CE Certification for VenusP-Valve™

Retrieved on: 
Friday, April 15, 2022
Venus, Patient, Marketing, Population, Percutaneous, Heart failure, Fluoroscopy, Tetralogy of Fallot, TAP, Hospital, NYHA, Evelina London Children's Hospital, Safety, Union, Medical imaging, EU

HANGZHOU, China, April 15, 2022 /PRNewswire/ --On April 8th, a Chinese company named Venus Medtech announced that its self-developed VenusP-ValvePercutaneous prosthetic pulmonary valve replacement system was approved by the European Union (CE) for marketing.

Key Points: 
  • HANGZHOU, China, April 15, 2022 /PRNewswire/ --On April 8th, a Chinese company named Venus Medtech announced that its self-developed VenusP-ValvePercutaneous prosthetic pulmonary valve replacement system was approved by the European Union (CE) for marketing.
  • VenusP-Valveis the first slef-expanding valve in Europe for the treatment of patients with right ventricular outflow tract disorder (RVOTD) following transannular repair (TAP).
  • In 2011, Venus Medtech invited international doctors to jointly develop VenusP-Valve.
  • In September 2016, Venus Medtech and Evelina London Children's Hospital officially launched the VenusP-Valve CE marking certification pre-market clinical trial.

The first treatment for sickle cell disease in over 20 years, Novartis' Adakveo®▼ (crizanlizumab) receives NICE recommendation for preventing recurrent vaso-occlusive crises

Retrieved on: 
Tuesday, October 5, 2021
People, Sickle cell disease, Recurrence, Guy's and St Thomas' NHS Foundation Trust, BMJ, Degenerative disease, Pain, VL, Health equity, Cell, United Kingdom, News, FAD, King, University College London Hospitals NHS Foundation Trust, Heart, Kidney, MAA, Twitter, SCD, Crizanlizumab, NICE, National Institute for Health and Care Excellence, P-selectin, Evelina London Children's Hospital, Partnership, Solution, Spleen, Sickle, NHS foundation trust, Volatile organic compound, Blood, Ageing, Life, Risk, Liver, National Institute, The New England Journal of Medicine, Society, NHR, Endothelium, Hope, Hospital, NHS England, King's College London, NHS, COVID-19, Research, Quality of life, Pharmaceutical industry, Vaccine

LONDON, Oct. 5, 2021 /PRNewswire/ -- Novartis is pleased to announce that eligible patients in England and Wales will soon have routine access to Adakveo®▼ (crizanlizumab) under a Managed Access Agreement (MAA). The news comes as the National Institute for Health and Care Excellence (NICE), published the Final Appraisal Determination (FAD) recommending crizanlizumab as an option for preventing recurrent sickle cell crises (two or more vaso-occlusive crises, VOCs, in a year, managed at home or in hospital) in people aged 16 or older with sickle cell disease (SCD).[2] Access to this innovative treatment not only helps with SCD but also demonstrates a commitment to supporting health equity for a community that has been historically underserved, without any new treatment options for more than 20 years, and disproportionately impacted by COVID-19.

Key Points: 
  • [4],[5] Sickle cell pain crises disrupt patients' lives physically, socially, and emotionally and can worsen into acute and long-term complications.
  • [3] While sickle cell disease mainly affects people of African or African-Caribbean origin, the sickle gene is found in all ethnic groups.
  • Kenny T and Tidy C. Sickle Cell Disease and Sickle Cell Anaemia patient information leaflet.
  • Management Strategies and Satisfaction Levels in Patients With Sickle Cell Disease: Interim Results From the International Sickle Cell World Assessment Survey (SWAY).