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

June 25, 2017
by inesp.alves
3 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
3 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

October 20, 2016
by inesp.alves
1 Comment

BioMarin presents report on phase 2 study of Vosoritide

BioMarin presented on the 19th October 2016 a press release on the data for Vosoritide in phase 2,  during the ASHG 2016 meeting. There is no overall novelty in this latest press release (19th Oct 2016) when comparing to the previous press … Continue reading

October 9, 2016
by inesp.alves
0 comments

Multidisciplinar assessments for achondroplasia by ALPE Foundation

ALPE Achondroplasia Foundation is based in Gijón, Asturias, north of Spain. It was founded in 2000 by Carmen Alonso, Miguel Lopez and the Press Lewis family. Now, the work done in the ALPE clinic is also accomplished by the support of James Healey. The … 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 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

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