Beyond Achondroplasia

Growing together with Clara

June 3, 2017
by inesp.alves
1 Comment

BioMarin updates the Multicenter and Multinational Clinical Study 111-901

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

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
1 Comment

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

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

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

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
1 Comment

BMN-111 – 6 key tidbits

Comments by Mark Schoenebaum, Biotech/Pharma analyst since 2000 MD, Johns Hopkins School of Medicine, 2000 BA, Indiana University, 1995 6 key tidbits:  1)      Phase 3 could enroll 50-100 patients and could be ~1 year in duration. Patient natural history could serve … 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

May 15, 2015
by inesp.alves
2 Comments

Quick BMN-111 update

BioMarin lauched on 30th April 2015 the latest investors report. An article from Benzinga.com said:”In the report, Cowen and Company noted, “BMN-111’s Phase II proof-of-concept study in achondroplasia is on track to produce data from first three dosing cohorts in … Continue reading

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