Beyond Achondroplasia

Growing together with Clara

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

April 14, 2016
by inesp.alves
3 Comments

LP Doc talk – About BMN-111

  Growing Stronger provides LP Doc talk, a series of online, live conversations with world-leading doctors and other experts focused on dwarfism. You can join live online for each session or visit the archived recordings anytime thereafter. This is a extremely … Continue reading

March 28, 2016
by inesp.alves
1 Comment

Patent and license agreement for CNP

Article credits: “the pharma letter” Chugai and BioMarin ink exclusive sublicense agreement   Japanese drug major Chugai Pharmaceutical (TYO: 4519) has concluded an exclusive sublicense agreement with US rare disease drug developer BioMarin Pharmaceutical (Nasdaq: BMRN) on the patent of Chugai’s … Continue reading

March 7, 2016
by inesp.alves
2 Comments

Roadmap for a new therapy – by BioMarin

BioMarin created a very clear and easy to read infographic poster about BMN-111, now called vosoritide. Here is just the first part, but it is worth seeing the complete infographic. It also explains the 4 different phases of a clinical trial. … Continue reading

February 2, 2016
by inesp.alves
2 Comments

Vosoritide Proceeding to Phase 3

On the 11th January 2016, at the 34th annual J.P. Morgan Healthcare Conference, Jean-Jacques Bienaimé, Chairman and Chief Executive Officer of BioMarin, did the following presentation: Recently secured “Freedom to Operate” worldwide Next Steps: 1. Pivotal Phase 3 Study  Health authority discussions … 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 19, 2015
by inesp.alves
1 Comment

New cohort revealed for phase 2 of BMN-111

The clinical trial: “A Phase 2 Study of BMN 111 to Evaluate Safety, Tolerability, and Efficacy in Children With Achondroplasia (ACH)” will have a new cohort of up to 60mg/kg. This information was obtain in the clinicaltrial.gov page. Cohort 4 has recently begun … 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

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