Beyond Achondroplasia

Growing together with Clara

August 15, 2018
by inesp.alves
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QED Tx – a new company working on a treatment for achondroplasia

At the beginning of 2018, QED Therapeutics was launched by Bridge Bio, as a new biotechnology company, focused on precision medicine for FGFR-driven diseases. 1. What are FGFRs? The fibroblast growth factor receptors (FGFRs) regulate important biological processes including cell proliferation … Continue reading

August 6, 2018
by inesp.alves
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Update: Focus groups with teenagers and parents- in the USA

There are updates for the new study to understand achondroplasia organized by Global Perspectives and will be conducted by ICON, a contract research organization, on behalf of a pharmaceutical company. This study has the purpose to: Understand the impact of … Continue reading

July 16, 2018
by inesp.alves
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Meclozine for achondroplasia heading to clinical trial

In February 2015, Matsushita et al., published “Meclozine Promotes Longitudinal Skeletal Growth in Transgenic Mice with Achondroplasia Carrying a Gain-of-Function Mutation in the FGFR3 Gene” (here in a short report in Beyond Achondroplasia) and shortly after in 2015, the same team also … Continue reading

June 9, 2018
by inesp.alves
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A way to contribute to the knowledge on achondroplasia

An ongoing study on pediatric achondroplasia conducted by the Brod Group is still enroling participants Call for participation – USA residents Parents with ACH with children with achondroplasia under 18 years-old children with ACH, with ages between 9 to 11 years-old … Continue reading

May 4, 2018
by inesp.alves
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The soluble FGFR3, Therachon´s TA- 46, may prevent early onset of obesity in achondroplasia

Obesity and metabolism are important topics related to achondroplasia but rarely discussed. Developed especially during childhood, obesity is a common complication associated with achondroplasia, affecting more than 50% of people with this condition. The extra weight exacerbates many other complications … Continue reading

August 25, 2017
by inesp.alves
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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
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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
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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
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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
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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

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