Beyond Achondroplasia

Growing together with Clara

September 4, 2016
by inesp.alves
2 Comments

RBM 007 – new approach for achondroplasia

The following news was presented in March 2016  by Fierspharma: Japan’s Agency for Medical Research and Development (AMED) named 8 projects for a pre-designation review as orphan drug commercialization candidates. These include achondroplasia therapy using anti-FGF2 aptamers from Tokyo-based Ribomic.   … 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

August 25, 2015
by inesp.alves
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B-701 – New potential treatment for achondroplasia

The motto of BioClin Therapeutics is “Developing protein therapeutics for high unmet medical needs”. And this has a deep meaning for achondroplasia. BioClin is a privately held biotechnology company funded in 2010, in California. Their leading product is B-701, that is … Continue reading

November 20, 2014
by inesp.alves
1 Comment

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

November 1, 2014
by inesp.alves
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Statins for achondroplasia update

I contacted Professor Noriyuki Tsumaki, the head researcher for the study of the use of statins in achondroplasia, also mentioned in a post here. I kindly got  the following asnwers: “Statins should not be used in children because statins decrease cholesterol, … Continue reading

September 18, 2014
by inesp.alves
5 Comments

Statins for achondroplasia?

Nature journal published yesterday an article connecting statins to achondroplasia treatment. You can read the abstract of the article “Statin treatment rescues FGFR3 skeletal dysplasia phenotypes“.A. Yamashita et al. Nature,doi:10.1038/nature13775, 2014 Statins or HMG-CoA reductase inhibitors are a class of drugs used … Continue reading

April 27, 2013
by inesp.alves
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FGFR3 music

When a pianist is playing a piano concert, one wrong key doesn´t put his career at risk. And just an attentive expert will notice the mistake. But just one “wrong key on our life concert”, and everything can sound completely … Continue reading

April 27, 2013
by inesp.alves
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Achondroplasia and riding a bike — Acondroplasia e andar de bicicleta

The cause of achondroplasia is now known for more than 10 years. However, all the efforts reunited until today didn´t translate into any effective therapy. Achondroplasia occurs due to a disturbance in bone growth process when it is mediated by cartilage. This process … Continue reading

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