October 23, 2017
by inesp.alves
2 Comments
The International Skeletal Dysplasia Society meeting was held in Bruges between the 20th and 23rd September 2017, with the presence of world-renowned geneticists and clinicians with interest in skeletal dysplasias; also some pharmaceutical companies and patients representatives from all around … Continue reading
October 20, 2017
by inesp.alves
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The following document, shared by BioMarin in September 2017, is a clarification on the ongoing clinical trials for achondroplasia and was released for associations and families: BioMarin Achondroplasia Programme Update 20Sept17 It is important to mention that families interested in … Continue reading
August 25, 2017
by inesp.alves
3 Comments
In 2016, the research team led by Dr. Pavel Krejci published the following article: Multikinase activity of fibroblast growth factor receptor (FGFR) inhibitors SU5402, PD173074, AZD1480, AZD4547 and BGJ398 compromises the use of small chemicals targeting FGFR catalytic activity … Continue reading
July 31, 2017
by inesp.alves
1 Comment
In 2014, several Reference Centers in France dedicated to diseases involving the same organs, created OSCAR, the French network of rare diseases of bone, calcium, and cartilage. Citing Dr. Geneviève Baujat (Necker-Enfants Malades hospital, Paris) “The aims are to expose … Continue reading
June 25, 2017
by inesp.alves
6 Comments
Therachon’s medicine in development for achondroplasia, TA-46, was granted Orphan Drug designation by the medicine agencies FDA and EMA. TA-46 is a type 3 fibroblast growth factor receptor antagonist addressing achondroplasia. This medicine is an inactive form of the receptor that … Continue reading
June 3, 2017
by inesp.alves
1 Comment
The “Multicenter, Multinational Clinical Assessment Study for Pediatric Patients With Achondroplasia” is a prospective observational study. This study’s code name is 111-901. The aim of this study is to collect systematic growth measurements of the children in order to collect data … Continue reading
May 14, 2017
by inesp.alves
3 Comments
BioCentury´s Senior writer Mike Leviten wrote a substantial article about the current medicines in research and development for achondroplasia. The article was published on the 7th April. This competition between pharma companies is very positive since it stimulates industry to … Continue reading
May 12, 2017
by inesp.alves
6 Comments
The following information is fully available at the European Union Clinical Trials Register. Some sections will be here highlighted: 1. Full title of the trial A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of BMN … Continue reading
April 9, 2017
by inesp.alves
3 Comments
A company based in Denmark and is applying its innovative TransCon technology that combines the benefits of prodrug and sustained-release technologies and developing the TransCon CNP for achondroplasia. FGFR3 and CNP Achondroplasia is caused by a gain-of-function mutation in fibroblast-growth-factor-receptor 3 (FGFR3). … Continue reading
February 13, 2017
by inesp.alves
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What is the central nervous system (CNS)? The nervous system has two parts: the central nervous system and the peripheral nervous system, due to their location in the body. The central nervous system (CNS) includes the nerves in the brain … Continue reading