Beyond Achondroplasia

Growing together with Clara

July 13, 2018
by inesp.alves
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A new step forward for TherAchon: the Dreambird study on Achondroplasia

TherAchon is a Biotechnology company that was established in 2014 and focuses on rare diseases. It is developing a drug for achondroplasia in particular: TA-46, which is planned to go onto phase I clinical trials in 2019 [1]. In order to … Continue reading

June 5, 2018
by inesp.alves
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A documentary on the lengthening process

The documentary  “The Lucas´s Journey“, by the film director Juan Enis, was produced by the mother of Lucas in 2015 and tells about the lengthening process of a boy with achondroplasia with 15 years-old, that had a total height of … Continue reading

March 4, 2018
by inesp.alves
3 Comments

5th Nordic Skeletal Dysplasia Symposium – full session dedicated to achondroplasia

The 5th Nordic Skeletal Dysplasia Symposium will be held in Copenhagen, on March 8-9, 2018. As stated in the Welcome note of this symposium “Skeletal dysplasias are rare diseases and the annual symposium is important to share medical knowledge with … Continue reading

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 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

June 25, 2017
by inesp.alves
6 Comments

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
1 Comment

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
6 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

January 5, 2017
by inesp.alves
5 Comments

Therachon protein for achondroplasia: TA-46

A press release from Business Wire stated that Therachon AG, a biotechnology company focused on rare genetic diseases, announced today it has appointed Luca Santarelli, M.D., as Chief Executive Officer and Director, and raised more $5 million for new medicines R&D (research a … Continue reading

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