Beyond Achondroplasia

Growing together with Clara

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

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

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

December 6, 2016
by inesp.alves
1 Comment

Assessing Health-related Quality of Life (HRQoL) in achondroplasia

What is quality of life? There is no single definition for Quality of Life, though there have been many attempts to define it. Similar to their definition of health, the World Health Organization’s (WHO) definition is among the most comprehensive … Continue reading

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

August 16, 2016
by inesp.alves
0 comments

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

June 30, 2016
by inesp.alves
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State of the art in achondroplasia

In the present, achondroplasia has more opportunities in the scientific search for a treatment than almost any other rare disease has. And this is very amazing. There are some relevant points in the science that is being produced for achondroplasia: … Continue reading

June 14, 2016
by inesp.alves
0 comments

Defective development and growth of the mandible in achondroplasia

An embryo is a new organism in the earliest stage of development. In humans this is defined as the developing organism from the fourth day after fertilization (when the union of a human egg and sperm cell occurs) to the end of … Continue reading

May 24, 2016
by inesp.alves
0 comments

New inhibitor of FGFR3 in mouse model

A therapeutic strategy to rescue dwarfism caused by FGFR3 gain-of-function mutations is to reduce or counteract the hyperactivity of FGFR3. Many cancers have FGFR3 mutations, in which the FGFR3 has hyperactivity, which contrast to the hyperactivity of FGFR3 in achondroplasia, that reduces … Continue reading

May 10, 2016
by inesp.alves
1 Comment

More about autophagy process in achondroplasia pathogenesis

Recently, in the AISAC congress (an italian association for the study and information on achondroplasia), a research project by TIGEM – Telethon Institute of Genetics and Medicine was presented who’s head researcher Prof. Carmine Settembre: “FGF signalling regulates bone growth through … Continue reading

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