Beyond Achondroplasia

Growing together with Clara

February 14, 2018
by inesp.alves
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Therachon starts phase 1 clinical trial with TA-46 for achondroplasia

Therachon announced today, 14 February 2018, the beginning of Phase 1 for the clinical trial with TA-46 for achondroplasia. This trial will take place in The Netherlands, with 70 adult healthy volunteers and it will be: randomized – A study in which … Continue reading

August 16, 2016
by inesp.alves
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The activity of FGFR3 with the achondroplasia mutation

Science is dynamic and new discoveries are emerging everyday. New advances on the activity of FGFR3, the receptor that has a mutation in achondroplasia, have been published. FGFs (fibroblast growth factors) and their receptors (FGFRs) play essential roles in tightly regulating … Continue reading

April 11, 2016
by inesp.alves
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ReACH Achondroplasia Registry

ReACH is a project created for achondroplasia by Prof. Krejci research team. “Prof. Krejci is doing research related to FGFR3 for more than 15 years, focusing in dissecting the mechanisms of fibroblast growth factor (FGF) signaling. His research team works … Continue reading

October 24, 2015
by inesp.alves
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The new link – autophagy in chondrocytes and achondroplasia

Why does, exactly, the mutant FGFR3 reduce so exaggeratedly chondrocyte proliferation in the growth plate? Well, there exists genetic information about achondroplasia since 1994. Francomano and Velinov found the location of the gene on the distal short arm of chromosome … Continue reading

October 1, 2015
by inesp.alves
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Soluble FGFR3 going forward with Therachon

Soluble FGFR3 was first mentioned here in September 2013. This protein is able to restore growth by acting has a decoy receptor and prevents FGFs from binding to mutant FGFR3 and was able to increase bone length and reduce complications associated with achondroplasia in transgenic … Continue reading

January 7, 2015
by inesp.alves
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Challenges of treating growth plate in children with achondroplasia

In “Achondroplasia: A view to the future options emerging from the benchside”, Narayana, J., Horton, W.A, 2013, there is a very interesting point that reflects the challenges of bringing FGFR3-based therapies to the bedside. “Despite the considerable recent progress in the … Continue reading

November 20, 2014
by inesp.alves
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CNP resistance in achondroplasia

Last month, while talking with Prof. Yayon (one of the world leaders in the research for a treatment for achondroplasia), I asked him about his opinion about the efficacy of BMN-111, and he answered: “Children with achondroplasia have lots of CNP”. Well, … Continue reading

October 17, 2014
by inesp.alves
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Achondroplasia highlights

During three full days, the V International Congress around Achondroplasia and other Skeletal Dysplasia took place in the north of Spain, very well-organized by Alpe Foundation. Several world-renowned experts attended, giving one or more lectures, over 500 people registered, mainly parents … Continue reading

August 3, 2014
by inesp.alves
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The paradox of FGFR3 signaling in skeletal dysplasia: why chondrocytes growth arrest while other cells over proliferate

A vision by P. Krejci of the reason why chondrocytes growth is less with FGFR3 mutation, at Mutation Research/Reviews in Mutation Research. 2014 Jan-Mar; 759:40-8. doi: 10.1016/j.mrrev.2013.11.001. Epub 2013 Dec 1. Somatic mutations in receptor tyrosine kinase FGFR3 cause excessive cell proliferation, leading … Continue reading

September 19, 2013
by inesp.alves
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New approach: postnatal soluble FGFR3

This past few weeks, have been rich in new acknowledgments around achondroplasia treatment. Yesterday, a research team from Inserm, published the following article: Postnatal Soluble FGFR3 Therapy Rescues Achondroplasia Symptoms and Restores Bone Growth in Mice   Abstract Achondroplasia is … Continue reading

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