Beyond Achondroplasia

Growing together with Clara

December 10, 2017
by inesp.alves
14 Comments

Update on BioMarin´s Achondroplasia Clinical Development Program

BioMarin Pharmaceutical sent to patient organizations an update of the clinical development program for BMN-111. The current status of the achondroplasia clinical programme is described in the document below. BioMarin’s investigational therapy for achondroplasia, BMN 111, is currently under investigation … Continue reading

May 12, 2017
by inesp.alves
6 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

November 23, 2016
by inesp.alves
1 Comment

A challenging report on Vosoritide

A rare disease encompasses any life-threatening or chronically debilitating disorder or condition which, as the name suggests, is uncommon in the general population. Rare diseases typically exhibit a high level of symptom complexity and as a result they very frequently require … Continue reading

October 20, 2016
by inesp.alves
3 Comments

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

April 21, 2016
by inesp.alves
6 Comments

Update in multicenter study from BioMarin

A Multicenter, Multinational Clinical Assessment Study for Pediatric Patients With Achondroplasia Eligibility Ages Eligible for Study: 2 Years to 13 Years Genders Eligible for Study: Both Accepts Healthy Volunteers: No Sampling Method: Probability Sample Study Population Approximately 200 patients will … Continue reading

April 21, 2016
by inesp.alves
3 Comments

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

November 25, 2015
by inesp.alves
1 Comment

BioMarin investors page announces

Vosoritide for achondroplasia In June, the Company published results from the Phase 2 study showing a 50 percent or 2.01 cm/year increase in mean annualized growth velocity (speed at which growth in children occurs) in the cohort of 10 patients … Continue reading

October 15, 2015
by inesp.alves
0 comments

BMN-111 phase 2 results presented at the ASBMR 2015 meeting

Dr. Melita Irving, the clinical geneticist who is coordinating the phase 2 clinical trial of BMN-111 in London, presented at the American Society for Bone and Mineral Research Annual 2015 Meeting (ASBMR) the initial six-month data from the first three … Continue reading

August 4, 2015
by inesp.alves
0 comments

BMN-111 Phase 3 ancillary cohort

BioMarin press release 3rd August 2015 “A major event in the second quarter of BioMarin Pharmaceutical Inc. was the completion and results of the Phase 2 study with vosoritide (future BMN-111 commercial designation) for the treatment of achondroplasia. BioMarin related that … Continue reading

June 18, 2015
by inesp.alves
2 Comments

Breaking news – BMN-111 Phase 2 proof-of-concept report

Vosoritide is the name we will know from now on. It’s the generic name of BMN-111 Report from the StreetInsider.com BioMarin announced positive results of a Phase 2 proof-of-concept and dose finding study of BMN 111 (vosoritide), an analog of … Continue reading

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