May 12, 2018
by inesp.alves
4 Comments
Ascendis Pharma announced on the 8th May it has dosed the first volunteers in a first-in-human phase 1 trial of TransCon CNP. (2) Ascendis TransCon technology includes the TransCon CNP that is a long-acting prodrug of a C-type natriuretic peptide … Continue reading
March 2, 2018
by inesp.alves
1 Comment
On March 22, 2018, FDA, the US Food & Drug Administration, will conduct a public advisory committee meeting on achondroplasia. The purpose of this meeting to discuss the major objectives of a phase 3 drug development program indicated for the … Continue reading
October 20, 2017
by inesp.alves
0 comments
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
6 Comments
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
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
6 Comments
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
3 Comments
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
0 comments
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
3 Comments
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
3 Comments
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