Beyond Achondroplasia

Growing together with Clara

May 13, 2016
by inesp.alves

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

February 21, 2016
by inesp.alves

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

January 20, 2015
by inesp.alves
1 Comment

BMN-111 clinical trial update

Recently, I had valid information that BioMarin announced that the measurement study is now open for recruitment again for the multicenter study. However, BioMarin didn´t change the age requirement, so children still need to be 4.5 years or older. The person … Continue reading

January 7, 2015
by inesp.alves

Challenges of treating growth plate in children with achondroplasia

In “Achondroplasia: A view to the future options emerging from the benchside”, Narayana, J., Horton, W.A, 2013, there is a very interesting point that reflects the challenges of bringing FGFR3-based therapies to the bedside. “Despite the considerable recent progress in the … Continue reading

November 20, 2014
by inesp.alves
1 Comment

CNP resistance in achondroplasia

Last month, while talking with Prof. Yayon (one of the world leaders in the research for a treatment for achondroplasia), I asked him about his opinion about the efficacy of BMN-111, and he answered: “Children with achondroplasia have lots of CNP”. Well, … Continue reading

November 18, 2014
by inesp.alves

Understanding clinical trials

This is a quite delicate subject once I´ve realized that many people, specially parents, see clinical trials as a way to convert their children or themselves into guinea pigs. Well, the goal of a clinical trial is to improve health … Continue reading

The first clinical trial and the modern trials

April 14, 2014 by inesp.alves | 2 Comments

At this point, BMN-111 is at phase 2 of a clinical trial, with the administration of this drug to around 20 children with achondroplasia over 5 years old.

This video is a very easy understandable explanation of what is a clinical trial, that helps to understand how to test which treatment might work best.

The history of clinical trials dates back to approximately 600 B.C. when Daniel of Judah conducted what is probably the earliest recorded clinical trial. He compared the health effects of the vegetarian diet with those of a royal Babylonian diet over a 10-day period. The trial had obvious deficiencies by contemporary medical standards (bias), but the report has remained influential for more than two millennia (H. Stolberg et al, Randomized Controlled Trials, 2004)

Modern clinical trials are randomized controlled. This is one of the simplest but most powerful tools of research. In essence, the randomized controlled trial is a study in which people are allocated at random to receive one of several clinical interventions.

Every year since 2005, the European Clinical Research Infrastructures Network (ECRIN) has celebrated the International Clinical Trials’ Day (ICTD) at or around the 20th of May ( to commemorate the day James Lind started his famous trial.

This May I will have the pleasure to attend to two important events to promote independent clinical research, promoted by the ECRAN project.
This event will promote a transnational communication on multinational clinical trials with the objective of facilitating better clinical research relevant to the needs of patients and highlight the important role of patients in clinical research.

Enjoy the video!

January 14, 2014
by inesp.alves

BMN-111 phase 2 – the first patient

January 14, 2014 Press release BioMarin Doses First Patient in Phase 2 Trial With BMN 111 for the Treatment of Children With Achondroplasia BioMarin Pharmaceutical announced today that it has dosed the first child in the Phase 2 trial with … Continue reading

December 17, 2013
by inesp.alves

Meclozine – one step closer

Meclozine is a drug in use for decades, and is used for motion sickness, without major adverse effects. And in this September, it became public that a japanese research team was studying meclozine to treat achondroplasia. Yesterday, I was given … Continue reading

May 18, 2013
by inesp.alves

BioMarin pharmaceutical – Achondroplasia clinical trial

After we had the genetic confirmation of Clara having achondroplasia, on the 2nd October 2012, I started to search about the subject. One week after that, I knew about the BioMarin´s clinical trial. At that moment, still without accepting her … Continue reading

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