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Pfizer Doses First Participants as Part of Global Achondroplasia Phase 2 Development Program

 
On December 15th, Pfizer announced that the first participants were dosed in the Global Phase 2 multiple dose, randomized study to assesses the safety, tolerability, pharmacokinetics, and efficacy of recifercept in children with achondroplasia.

Participants received a subcutaneous injection of the biologic recifercept at Antwerp University Hospital, Belgium, under the care of Professor Geert Mortier, and Hospital Vithas San José, Spain under Dr.Josep De Bergua. 

 

Read the press statement here

Publication


Biallelic cGMP-dependent type II protein kinase gene (PRKG2) variants cause a novel acromesomelic dysplasia.


It has been recently published an article describing the clinical, radiological and genetic aspects of a novel acromesomelic dysplasia. Two centres involved, Hospital Universitario la Paz, Spain and Sheffield Childrens NHS foundation Trust, UK are members of ERN BOND.
Read the article here

Webinars 


ERN BOND contributes to the Rare Disease Day  by organizing a series of webinars about rare bone diseases, together with some Italian patient associations - As.It.O.I., A.C.A.R. and UNIAMO FIMR - and with the patronage of Fondazione Telethon

Note that all the webinars will be in Italian only.


 


January 21st, 7 pm CET:
 
COVID-19: trattamento farmacologico e di supporto per pazienti affetti da OI
(COVID-19 and indications for patients with OI)
  • dr. Andrea Vianello, Università di Padova
  • dr. Luca Sangiorgi, Istituto Ortopedico Rizzoli, Bologna   
Join Zoom Meeting: https://us02web.zoom.us/j/81332784559?pwd=ZzdqMUJhL0J0V29YaGNSTVFENGN3Zz09 

Meeting ID: 813 3278 4559
Passcode: 281583

 
Find your local number

January 28th, 7 pm CET:

COVID-19 e vaccini
(COVID-19 and vaccines)
  • prof. Ivan Gentile, Università Federico II Napoli
  • dr. Luca Sangiorgi,  Istituto Ortopedico Rizzoli, Bologna 
 
February 22nd, 7 pm CET:

 COVID-19 e emergenza psicologica
(COVID-19 and psychological impact)
  • dr. Manila Boarini, Istituto Ortopedico Rizzoli, Bologna
  • dr. Marianna Matera, As.It.O.I

March 4th, 7 pm CET:

Tools per il follow-up a distanza dei pazienti affetti da malattie rare scheletriche
(Tools for remotely following up patients with rare skeletal diseases)
  • prof. Gianluca Moro, Università di Bologna
  • dr. Luca Sangiorgi, Istituto Ortopedico Rizzoli, Bologna
  • dr. Paolo Fraschini, IRCCS Eugenio Medea, Bosisio Parini 
Links to attend the webinars will be uploaded here

Endocrine and bone management in Duchenne muscular dystrophy

 

Endo-ERN organised a webinar on Duchenne muscular dystrophy (DMD), a rare X-linked inherited neuromuscular condition affecting 1 in 4000 boys.

In this webinar Dr. Jarod Wong, from the University of Glasgow, will review the endocrine and bone morbidity in DMD with focus on the updated care recommendations and discuss areas for future research. He will be joined by Justus Kuijer who will share his experience of endocrine and bone morbidity.

The webinar will be held on January 25th at 2:30 pm CET.

 
Register here to attend the webinar

Launch of Exchange Programme for healthcare professionals in European Reference Networks


Knowledge sharing and stimulating collaboration between health care professionals in ERNs. That is the aim of the Exchange Programme 2020-2022, funded by the European Commission. In the coming two years, three different rounds of exchanges will take place between professionals of HealthCare Providers that are members or affiliated to an ERN.
 
The objective of the ERN Exchange Programme is to palliate disparities in specific knowledge or gaps in expertise by facilitating the arrival of high-level expertise in a considerable number of diseases to a big number of Healthcare Providers.
 

Ecorys Ltd will provide services to all ERNs in the framework of the Exchange Programme of the European Commission. More information on the Exchange Programme will follow soon.

The Internal Call for Innovation Project in Clinical Trials Methodology in Limited Populations


The Internal Call for Innovation Project in Clinical Trials Methodology in Limited Populations has been launched on December 7, 2020. The call is open to EJP RD beneficiaries and their linked third parties.
 

 The innovation methodologies topics particularly include (but are not limited to): 
 
  • Development of a disease progression model from a natural
    history cohort or other observational studies.

     
  • Development and validation of a disease specific clinically meaningful
    outcome with special interest in PCOMs, or composite endpoints.

     
  • Development of a design and analysis procedure for a
    pharmacometric model and/or bridging study.

     
  • Development of a randomization-based model as an
    alternative analysis strategy and explore the level of evidence.

     
The call aims to encourage collaborations among groups of experts consisting of different stakeholders including methodological experts, clinicians, patients and industry (when relevant), who will jointly develop innovative ready-to-use methods to enhance RD clinical trial methodologies.

Foreseen call closing deadline is 3 March 2021.
 
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ERN BOND · Istituto Ortopedico Rizzoli · Via di Barbiano, 1/10 · Bologna, BO 40136 · Italy

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