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

May 12, 2017
by inesp.alves
3 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

January 5, 2017
by inesp.alves
5 Comments

Therachon protein for achondroplasia: TA-46

A press release from Business Wire stated that Therachon AG, a biotechnology company focused on rare genetic diseases, announced today it has appointed Luca Santarelli, M.D., as Chief Executive Officer and Director, and raised more $5 million for new medicines R&D (research a … Continue reading

September 4, 2016
by inesp.alves
2 Comments

RBM 007 – new approach for achondroplasia

The following news was presented in March 2016  by Fierspharma: Japan’s Agency for Medical Research and Development (AMED) named 8 projects for a pre-designation review as orphan drug commercialization candidates. These include achondroplasia therapy using anti-FGF2 aptamers from Tokyo-based Ribomic.   … 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 25, 2015
by inesp.alves
0 comments

B-701 – New potential treatment for achondroplasia

The motto of BioClin Therapeutics is “Developing protein therapeutics for high unmet medical needs”. And this has a deep meaning for achondroplasia. BioClin is a privately held biotechnology company funded in 2010, in California. Their leading product is B-701, that is … Continue reading

August 9, 2015
by inesp.alves
0 comments

Statins for achondroplasia, at the 12th ISDS meeting -part 1

The ISDS is registered as a non-profit organization, and the principal aim of the Society is to promote scientific progress in the field of skeletal dysplasias and dysostoses. At the Nosology and classification of genetic skeletal disorders, Superti-Furga et al. 2011: “Dysostoses are disorders affecting … 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

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