Beyond Achondroplasia

Growing together with Clara

April 20, 2014
by inesp.alves

Stages of implementation of BMN-111 in a short summary

BMN 111 Clinical Studies in Achondroplasia (information by: BioMarin)   1-Double-Blind, Placebo-Controlled Phase 1 Study (Protocol 111-101) Results of a two-part, double-blind, placebo-controlled Phase 1 study (Protocol 111-101) conducted in 48 healthy adult volunteers demonstrated that systemic exposure to BMN … 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!

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