Beyond Achondroplasia

Growing together with Clara

October 20, 2017
by inesp.alves
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Recruitment in clinical trials

The following document, shared by BioMarin in September 2017, is a clarification on the ongoing clinical trials for achondroplasia and was released for associations and families: BioMarin Achondroplasia Programme Update 20Sept17 It is important to mention that families interested in … Continue reading

June 25, 2017
by inesp.alves
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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
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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 12, 2017
by inesp.alves
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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

October 20, 2016
by inesp.alves
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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

May 17, 2016
by inesp.alves
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Questions and answers – BMN-111 Drug Trial

LP Doc Talk is a project of Growing Stronger, a foundation based in S. Francisco, California and founded by Amer and Munira Haider, parents of Ahmin,a boy now with 7 years-old, born with achondroplasia. When I started to search about achondroplasia, I … Continue reading

May 13, 2016
by inesp.alves
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BMN-111 Phase 2: study 111-202 and extension study 111-205

Study 111-202 A Phase 2 Study of BMN 111 to Evaluate Safety, Tolerability, and Efficacy in Children with Achondroplasia (ACH) This study is enrolling participants by invitation only   Estimated Enrollment: 46 Study start date: January 2014 Estimated Study Completion … Continue reading

April 21, 2016
by inesp.alves
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Vosoritide for Achondroplasia – Rare disease day 20th April 2016

On the 20th April at 8am, NY time, Sarah Noonberg, MD, PhD, Group Vice President, Head of Global Clinical Development, presented the updates for Vosoritide. List of presented updates: 10 children, aged 6 to 11, received for 12-month a dose of 15 μg/kg/day (cohort … Continue reading

March 4, 2016
by inesp.alves
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Science with no results shared

One very important issue around clinical trials and many research projects (many are funded by public money) is that, at the end, the data and results are not published. In the site clinicaltrials.gov, when searching for studies in achondroplasia, it … Continue reading

February 21, 2016
by inesp.alves
2 Comments

Achondroplasia Natural History Multicenter Clinical Study

The title of this article is the designation of a study for achondroplasia. The novelty of this study is the attempt of doing a global registry for patients with achondroplasia. But what is the purpose of a patients registry? A registry … Continue reading

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