30th March 2021

Newsletter March 2021

The recent approval by regulatory agencies worldwide of Zolgensma, a gene therapy for children with type 1 Spinal Muscular Atrophy 1 (SMA1), raised happiness and gratitude across families and scientific communities.
SMA1 is a genetic neuromuscular disorder that affects the nerve cells that control voluntary muscles (motor neurons). Without treatment, symptoms of SMA1 become apparent before 6 months of age and include worsening muscle weakness and poor muscle tone (hypotonia) due to loss of the lower motor neurons in the spinal cord and brain stem.
Feeding and breathing problems are also present. SMA1 is caused by changes (pathogenic variants also called mutations) in the SMN1 gene and is typically inherited in an autosomal recessive manner. Zolgensma, which is manufactured by Novartis Gene Therapies (a division of Novartis Pharmaceuticals), has been shown to help babies with breathing without a ventilator, sitting up on their own, crawling and walking after a single infusion treatment; those are very relevant milestones when it comes to their quality of life.
The latest data suggest that Zolgensma can provide rapid and sustained improvement in motor functions for young children with type 1 SMA and prolong their lives. CVBF is committed to paediatric research and supports the development of innovative therapies and drugs for the treatment of rare diseases affecting children, such as SMA1.
“We will put all our efforts and scientific expertise to make a difference in the lives of these children to provide them all treatments they deserve. It is time to overcome barriers that prevent access to innovative treatments, and to change the approach of funders and policymakers by considering the real value of disability-adjusted life years, together with the loss of productivity of the caregivers and the expenses of the sanitary services”, said Donato Bonifazi, CVBF CEO.


cASPerCF study can start recruiting

We are happy to announce you that the cASPerCF study has opened the first two sites participating in the Study and can now start the patient’s recruitment. The first Site Initiation Visit (SIV) was held on February 12th, 2021 at the Ospedale Pediatrico Bambino Gesù (OPBG) in Rome, followed by the second SIV held on March 9th, 2021 at the Azienda Socio Sanitaria Territoriale degli Spedali Civili in Brescia.
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PedCRIN webinar series


From April 7th to June 16th the PedCRIN project is launching a webinar series to share some of the most interesting outcomes of the project. The webinars are aimed to present the tools that have been developed to facilitate the execution of clinical trials for neonates, children and young people. On April 7th 2021, Viviana Giannuzzi from Gianni Benzi Foundation and Cristina Manfredi from CVBF, PedCRIN’s Partner, will explain the tools for the management of paediatric trials.
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The Current Status of European Research related to COVID-19

To face COVID-19 pandemic, many hospitals have been forced to drastically change their organisation in order to find as much space as possible to accommodate COVID-19 patients; this situation has created an organisational chaos as regards both to therapies for patients not affected by COVID-19 and the stand-by of Clinical Trials (CTs) activities.
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Clinical Trials Information System (CTIS): training programme

EMA has recently launched the CTIS training webpage for all user groups and organisations to facilitate their preparedness. This page contains information about the CTIS training programme and the online self-study material catalogue as foreseen by the CTIS training strategy. On April 26th from 10.00 to 11.30 am EUCROF Events&Training working group, chaired by Donato Bonifazi, is organising the webinar on the EMA Clinical Trials Information System.
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Diagnosis and Treatment of Chronic Neuropathic and Mixed Pain in Children

The articleDiagnosis and Treatment of Chronic Neuropathic and Mixed Pain in Children and Adolescents: Results of a Survey Study amongst Practitioners” developed within the GAPP project has been published in Children open access journal. The article provides an evidence of the international practice amongst practitioners for the diagnosis and treatment of chronic, neuropathic pain in children and adolescents.
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PedCRIN survey submitted to Contemporary Clinical Trials 

We are pleased to announce that the collaborative work developed within the PedCRIN project on the survey administered to capture the needs to receive infrastructural support to cover specific paediatric research gaps, has been accepted for publication in Contemporary Clinical Trials Communications journal.
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SAVE THE DATE: Technical workshop on real-world metadata for regulatory purposes

The HMA-EMA Big Data Steering Group is pleased to announce the upcoming virtual workshop focused on “data characterization and discoverability” that will be held on Monday, 12 April 2021. The HMA-EMA joint Big Data Task Force recommended “to promote data discoverability through the identification of metadata” as part of its Recommendation III, which is reflected in the Big Data Steering Group work plan.
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About the Author: Flogert Dollani


By Published On: March 30th, 2021Categories: Newsletter0 Comments