Beyond Achondroplasia

Growing together with Clara

October 23, 2017
by inesp.alves
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The International Society of Skeletal Dysplasia 2017 meeting report

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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Recruitment in clinical trials

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

Research on ARQ 087, a Tyrosine kinase inhibitor

  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
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Exercises and postural education for children with achondroplasia – OSCAR 2016

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
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Therachon heading to Phase 1 clinical trial

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
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BioMarin updates the Multicenter and Multinational Clinical Study 111-901

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 published the article “Competing for growth”

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
4 Comments

Phase 3 trial for achondroplasia – BioMarin Study 111-301

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
1 Comment

The TransCon CNP, a prodrug for achondroplasia

    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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Foramen magnum growth in achondroplasia

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

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