brains4brain | Brains for Brain

27/11/2019

14th Brains for Brain Foundation (B4B) International Workshop, 6th -8th March 2020, Frankfurt – Germany

We are pleased to announce the forthcoming 14th meeting of the Brains for Brain Foundation (B4B).

B4B is an International Task Force (chaired by Prof Maurizio Scarpa) composed of outstanding scientists and ‘clinicians’ who are leaders in the neurological field. Together with Biotech companies they aim to facilitate joint efforts to improve the understanding of rare genetic diseases affecting and seriously damaging the brain, in particular in children. The main mission of the fundation infact consists in the funding of translational research and education in the field of neurodegenerative paediatric disorders mainly focusing the attention on a group of about 40 genetically and biochemically very well characterised diseases, called Lysosomal Storage Disorders (LSDs).

According to its traditional programme, the workshop will be held in Frankfurt, Germany, since this venue is really well connected with all the countries and facilitates the participations of people from different parts of the world.

The meeting will therefore take place at the Hotel Mercure Frankfurt Airport.

Please note that this year, to avoid overlapping with other important meetings, this year we will have it in March.

The meeting will in fact begin on March 6th at 2pm and end on the 8th with lunch.

As usual, the programme will contain scientific and strategic sessions in which the following issues wlil be discussed:

1. Research achievements in the field of neurodegenerative diseases, in particular lysosomal storage disorders (LSDs) at clinical and basic science level and in the field of Blood Brain Barrier;

2. New recent advances on natural history and pathophysiology of LSDs;

3. Factors which control the entry into the brain of medicines and other therapeutic agents which may be helpful in treating central nervous system disease;

4. How B4B, the European Reference Network on Rare heridary diseases (MetabERN) and the European Union might collaborate to stimulate interest on research in rare diseases;

5. The role of the industries in driving innovation for new therapeutic approaches for true unmet needs.

A draft of the scientific programme is available here.

If you wish to attend the workshop, please get in touch with Mrs Paula Caprelli (p.caprelli@jazzitaly.com) as soon as possible in order for us to be able to register your presence and predispose the logistics.

For general enquiries or any additional required information, please get in touch with Dr. Cinzia Maria Bellettato (c.bellettato@gmail.com)

28/02/2019

FEBRUARY 28 2019, IT’S THE RARE DISEASE DAY

by brains4brain

mugs-r7bbfd3a5b7f74d7eb22d000b017967bd_x7jgr_8byvr_500

Today is Rare Disease Day: it is time to ‘show your rare’

Senza categoria

28/02/2019

CONTRIBUTIONS FROM ITALIAN PUBLIC INSTITUTIONS

by brains4brain

In accordance with the relevant law (124/2017) of the Italian civil code, we hereby publish the information regarding the contributions we received from Italian public institutions during 2018.

(Only Italian)

COMUNICAZIONE AI SENSI ART. 1 COMMI 125 – 129 LEGGE 04.08.17 N. 124

La legge 124/2017 (pubblicità e trasparenza) ha introdotto, per gli Enti del Terzo Settore, l’obbligo di pubblicare entro il 28 febbraio di ogni anno sul proprio sito internet (o sulla pagina facebook) le informazioni relative a “sovvenzioni” – “contributi” – “incarichi retribuiti” o comunque “vantaggi economici” di qualunque genere incassati nell’anno precedente e provenienti da pubbliche amministrazioni o da soggetti equiparati.

Nel rispetto di tale legge pubblichiamo di seguito il prospetto contabile della Fondazione B4B relativo all’anno 2018

Soggetto erogatore

Ammontare

Data incasso

Causale

Ministero del Lavoro e delle Politiche Sociali

4.254,62

16.08.2018

Contributo 5×1000

(annualità 2015-2016)

Senza categoria

12/02/2019

PRECEPTORSHIP PROGRAMME ON RARE DISEASES: MUCOPOLYSACCHARIDOSIS, MANAGEMENT AND TREATMENT – Salzburg, April 10th, 11th and 12th, 2019

by brains4brain

The Brains for Brain Foundation, in collaboration with the Clinical Research Center in Salzburg and the Medizinisches Simulationszentrum Salzburg, and as part of the capacity building and training activities of the European Reference Network for hereditary Metabolic disorders (MetabERN), has organized a PRECEPTORSHIP PROGRAMME ON RARE DISEASES: MUCOPOLYSACCHARIDOSIS (MPS), MANAGEMENT AND TREATMENT” that will take place at the Salzburg Medical Simulation Center on April 10^th, 11^th and 12th, 2019.

The course will guest about 15 physicians from different countries who will visit to share experiences and being updated on the recent development on MPS. The meeting will also provide a comprehensive global forum for experts and participants to exchange ideas and present results on all aspects of MPSs.

For more details please see the complete programme.

Objective – The overall objective of the course is to train professionals with a scientific background and an interest on Lysosomal Storage Diseases, in particular Mucopolysaccharidoses (MPSs) and to provide them with theoretical expert knowledge and practical experience in both the recognition of these diseases and related management. The practical experience in management and treatment of MPSs will be gained through the use of patient case studies that the participants will work on throughout the course and in parallel with new topics being added to their theoretical knowledge base.

Expected results – The main expected results of this program consist in ensuring safe and effective initiation of therapies in patients from countries where there is not experience of administration; decreasing the possibility of severe adverse events during treatment and helping patients to receive a state of the art follow up.

Learning outcomes – At the end of the course participants will be able to discuss a multidisciplinary approach for the management and treatment of these complex disorders assuring an adequate follow up of patients by evaluating the status of the disease.

Participants and Requirements – Participants (max 15) are expected to have quite a good experience in the management of MPS patients in particular with the ERT and its possible adverse events. The Preceptorship will be run in English, a good knowledge of the English language is mandatory. Participants are required to attend for the entire length of the preceptorship.

How to apply – If you are interested in the PRECEPTORSHIP PROGRAMME ON RARE DISEASES: MUCOPOLYSACCHARIDOSIS, MANAGEMENT AND TREATMENT, please send an email with your CV to Dr. Cinzia Maria Bellettato, the Brains for Brain Scientific Officer, at c.bellettato@gmail.com

All candidates will be evaluated according to the skills, qualifications, experience and professional interest considered necessary to successfully complete the preceptorship.

IMPORTANT: Please note that B4B will cover travel from EU countries only and that non-EU affiliates will have to cover for flights.

For more details please see the complete programme.

Brains4Brain | Senza categoria | Training Programme

25/01/2019

13th Brains for Brain Foundation (B4B) International Workshop, 24th -26th January 2019, Frankfurt – Germany

by brains4brain

Prof Maurizio Scarpa (IT) and Dr David Begley (UK) have officially opened yesterday the 13th meeting of the Brains for Brain Foundation (B4B) that is taking place in Frankfurt in these days (January 24th-26th, 2019)

This last edition brought together about 100 outstanding scientists and ‘clinicians’ leaders in the neurological field. Such an extended participation is a sign of the increasing awareness of the importance of collaborative research and interdisciplinary dialogue among experts working on the neurological rare diseases field. In particular the active participation of new and old attendees seriously committed to combine their forces and their scientific experiences is a proof of evidence of the successful networking activity performed by the Brains for Brain Foundation.

B4B is an International Task Force (chaired by Prof Maurizio Scarpa) aimed at funding translational research and education in the field of neurodegenerative paediatric disorders, mainly focusing the attention on a group of about 40 genetically and biochemically very well characterised diseases, called Lysosomal Storage Disorders (LSDs).

The scientific programme is available HERE.

Senza categoria

20/08/2018

13th Brains for Brain Foundation (B4B) International Workshop, 24th -26th January 2019, Frankfurt – Germany

by brains4brain

We are pleased to announce the forthcoming 13th meeting of the Brains for Brain Foundation (B4B).

The meeting will therefore take place at the Hotel Mercure Frankfurt Airport.

Please note that this year, to avoid overlapping with other important meetings (e.g the Gordon Lysosomal Disease Conference in Galveston, Texas, March 3rd -8th 2019), we will have it in January.

The meeting will in fact begin on January 24th at 2pm and end on the 26th with lunch.

As usual, the programme will contain scientific and strategic sessions in which the following issues wlil be discussed:

1. Research achievements in the field of neurodegenerative diseases, in particular lysosomal storage disorders (LSDs) at clinical and basic science level and in the field of Blood Brain Barrier;

2. New recent advances on natural history and pathophysiology of LSDs;

3. Factors which control the entry into the brain of medicines and other therapeutic agents which may be helpful in treating central nervous system disease;

4. How B4B, the European Reference Network on Rare heridary diseases (MetabERN) and the European Union might collaborate to stimulate interest on research in rare diseases;

5. The role of the industries in driving innovation for new therapeutic approaches for true unmet needs.

A draft of the scientific programme will be made available HERE.

For general enquiries or any additional required information, please get in touch with Dr. Cinzia Maria Bellettato (c.bellettato@gmail.com)

Senza categoria

10/03/2018

Launch of a new public-private partnership to improve clinical trial infrastructure to facilitate the development of new drugs for children in Europe

by brains4brain

The members of the “conect4children” (c4c) initiative today announced the start of a large
collaborative paediatric network that will facilitate the development of new drugs and other
therapies for the entire paediatric population in Europe.

The conect4children (collaborative network for European clinical trials for children, (c4c) consortium aims to enhance the competitiveness of Europe as a critical region for developing medicines for children by using existing expertise, patient access and developing common processes to be applied to disease natural history studies, registries, studies of new therapies and comparisons of existing therapies.
The consortium is a novel collaboration between academic and private sectors that includes 33 academic and 10 industry partners from 20 European countries, more than 50 third parties and around 500 affiliated partners.
The six-year project, comprised of a multidisciplinary public-private consortium, brings together key stakeholders across academia and industry. It is a pioneering opportunity to build capacity for the management of multinational paediatric clinical trials across Europe whilst ensuring the voices of children, young people and their families are heard. Strong links with regulators will be established.
There are many scientific and operational challenges faced by both pharmaceutical companies and academia when running paediatric clinical trials. According to Prof. Carlo Giaquinto of Fondazione PENTA Onlus and University of Padova, who coordinates the project, “c4c will address critical problems with the design, implementation and
operational conduct of paediatric clinical trials, such as fragmented and redundant efforts between sponsors, sites and countries; the paucity of patients available for study in many paediatric indications and the need for multiple capable sites and expertise to make trials successful.”

This project aims to generate a sustainable infrastructure that optimises the delivery of clinical trials in children through:
• a single point of contact for all sponsors, sites and investigators
• efficient implementation of trials adopting consistent approaches, aligned quality standards and coordination of sites at national and international level
• collaboration with specialist and national networks
• high quality input to study design and preparation through rigorous strategic and operational feasibility assessment
• the promotion of innovative trial design and quantitative science methods
• an education and training platform to shape the future leaders of paediatric drug development
• the development of sustainable support for all these activities

One of the key goals of the project is to support the use of innovative trial designs and new quantitative methods to foster development of new innovative medicines and to support development in rare paediatric diseases and high medical need area “Children must have access to innovative medical therapies that have been developed with the same degree of urgency and rigor as those for adults,” said Joanne Waldstreicher, MD, Chief Medical Officer, Johnson & Johnson. “With conect4children in Europe joining in this effort, complementing work under way with I-ACT for Children in the United States, we will be able to accelerate the availability of high quality scientific data
that can improve the safe and effective use of therapies in children.”
“Clinical trials with medicinal products for paediatric use are one of the most sensitive areas in science – both from a medical and an ethical perspective”, said Dr. Michael Devoy, Chief Medical Officer of Bayer. “Improving the clinical trial infrastructure is an import step in enabling children to take part in medical progress”.
Dr. Mark Turner, Co-coordinator of the project, University of Liverpool, stated: “This network will have a significant impact on how we develop much-needed innovative and improved medicines for babies, children and young people. A number of collaborations built up over the past decade will contribute to this pan-European research network. The
University of Liverpool is proud to be collaborating with institutions and research networks across Europe”
With a budget of about 140 Mio€ (IMI2 support of 67 Mio€ and industry partners’ in-kind contribution of 73 Mio€), c4c is one of the biggest initiatives funded by the Innovative Medicines Initiative 2 Joint Undertaking (IMI2 JU) under grant agreement n º 777389. The Innovative Medicines Initiative 2 Joint Undertaking is Europe’s biggest Public Private Partnership and is funded jointly by the European Union´s Horizon 2020 research and
innovation programme and the European pharmaceutical industry (represented by EFPIA, the European Federation of Pharmaceutical Industries and Associations).

• More info on IMI: www.imi.europa.eu
• Follow IMI_JU on Twitter: @IMI_JU

The parties involved
The project is coordinated and led by: Fondazione PENTA – for the treatment and care of children with HIV – ONLUS, The University of Liverpool, Janssen Pharmaceutica NV, Bayer AG Other partners are: Ospedale Pediatrico Bambino Gesù; EURORDIS – European Organisation for Rare Diseases Association; European Cystic Fibrosis Society; Stichting Katholieke Universiteit; Swiss Clinical Trial Organisation Verein; Associação para Investigação e Desenvolvimento da Faculdade de Medicina; Istituto Giannina Gaslini; University College London; SIOP Europe ASBL; Tartu Ulikool; Okids GMBH; University of Newcastle upon Tyne; Universiteit Gent; Universitaetsklinikum Heidelberg; Aristotelio Panepistimio Thessalonikis; Instytut Pomnik Centrum Zdrowia Dziecka; Helse Bergen
HF*Haukeland University Hospital; ECNP Research & Scholarship Foundation; Robert Bosch Gesellschaft fur Medizinische Forschung MBH; University College Cork– National University of Ireland, Cork; Karolinska Institutet; Fundacio Sant Joan de Deu; Servizo Galego de Saude; Gyermekgyógyászati Klinikai Vizsgálói Hálózat; Fondazione per la Ricerca Farmacologica Gianni Benzi Onlus; ECRIN European Clinical Research Infrastructure Network; The Hospital District of Helsinki and Uusimaa; Institut National de la Sante et de la Recherche Medicale; HSK DR Horst Schmidt Kliniken Wiesbaden Gmbh; ARSENAL.IT-Centro Veneto Ricerca e Innovazione per la Sanità Digitale; Univerzita Karlova; Sanofi-Aventis Recherche & Développement; Eli Lilly and Company Limited; UCB Biopharma SPRL; Novartis Pharma AG; Institut de Recherches Internationales Servier; GlaxoSmithKline Research and Development LTD.; Pfizer Limited; F. Hoffmann – La Roche AG

The full list of organisations involved in the project can also be found at the c4c webpage
www.conect4children.org

Project Office/General Enquires: Email us. communication@conect4children.org

Disclaimer This communication reflects the views of the c4c Consortium and neither IMI nor the
European Union and EFPIA are liable for any use that may be made of the information
contained herein
H2020-JTI-IMI2-2016-10. Proposal: 777389

Senza categoria

28/02/2018

FEBRUARY 28 2018, IT’S THE RARE DISEASE DAY

by brains4brain

Today is Rare Disease Day: it is time to ‘show your rare’

Senza categoria

13/02/2018

SSIEM Annual Symposium

by brains4brain

Brains for Brain is pleased to inform you that the Annual Symposium of the Society for the Study of Inborn Errors of Metabolism (SSIEM) will be held in Athens from September 4th to September 7^th 2018.

The theme of the scientific program is: “Old Roads, New Connections”. The objective of the symposium is to show how ‘OLD’ knowledge is being transformed and ‘NEW CONNECTIONS’ are being recognized, which help us to see the bigger picture, understand the physiology and thus the pathophysiology of IEM’s and provide new diagnostic tools and new targets for treatment.

For more infor please see http://www.ssiem2018.org/.

Senza categoria

02/02/2018

12th Brains for Brain Foundation (B4B) International Workshop, 8-10 March 2018, Frankfurt –Germany

by brains4brain

We are pleased to announce the forthcoming 12th meeting of the Brains for Brain Foundation (B4B) .

According to its traditional programme, the workshop (March, 8th to 10th, 2018) will be held at the Lindner Congress Hotel in Frankfurt, Germany, since this venue is really well connected with all the countries and facilitates the participations of people from different parts of the world.

As usual, the programme will contain scientific and strategic sessions in which the following issues wlil be discussed:

1. Research achievements in the field of neurodegenerative diseases, in particular lysosomal storage disorders (LSDs) at clinical and basic science level and in the field of Blood Brain Barrier;

2. New recent advances on natural history and pathophysiology of LSDs;

3. Factors which control the entry into the brain of medicines and other therapeutic agents which may be helpful in treating central nervous system disease;

4. How B4B and the European Union might collaborate to stimulate interest on research in rare diseases,

5. The role of the industries in driving innovation for new therapeutic approaches for true unmet needs.

You can find the scientific programme here.

For general enquiries or any additional required information, please get in touch with Dr. Cinzia Maria Bellettato (c.bellettato@gmail.com)

Senza categoria

All posts by brains4brain

Today is Rare Disease Day: it is time to ‘show your rare’

Today is Rare Disease Day: it is time to ‘show your rare’