Beyond Achondroplasia

Growing together with Clara

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

January 20, 2015
by inesp.alves
1 Comment

BMN-111 clinical trial update

Recently, I had valid information that BioMarin announced that the measurement study is now open for recruitment again for the multicenter study. However, BioMarin didn´t change the age requirement, so children still need to be 4.5 years or older. The person … Continue reading

November 20, 2014
by inesp.alves
1 Comment

CNP resistance in achondroplasia

Last month, while talking with Prof. Yayon (one of the world leaders in the research for a treatment for achondroplasia), I asked him about his opinion about the efficacy of BMN-111, and he answered: “Children with achondroplasia have lots of CNP”. Well, … Continue reading

November 9, 2014
by inesp.alves
0 comments

World Orphan Drug Congress Europe 2014

Europe’s go-to event for orphan drug developers, patient groups and payers. The conference program is vast. There will be several roundtable sessions on key issues in the Orphan and Rare Disease Sector as Patient Focused Sessions. At the roundtable sessions, leading providers … Continue reading

October 17, 2014
by inesp.alves
2 Comments

Achondroplasia highlights

During three full days, the V International Congress around Achondroplasia and other Skeletal Dysplasia took place in the north of Spain, very well-organized by Alpe Foundation. Several world-renowned experts attended, giving one or more lectures, over 500 people registered, mainly parents … Continue reading

September 5, 2014
by inesp.alves
5 Comments

5th International Congress around achondroplasia and other skeletal dysplasias

Alpe Foundation, one of the most important spanish and european organisations for achondroplasia, is promoting  the 5th Congress, that will take place in north of Spain, Gijon, on the 11th, 12th and 13th October 2014. Their webpage is in spanish … Continue reading

June 17, 2014
by inesp.alves
2 Comments

BioMarin at the Wells Fargo 9th Annual Healthcare Conference

Dan Oppenheimer, Group Vice President, Development & Portfolio Strategy – BioMarin Pharmaceutical Inc, presented today at the Wells Fargo research conferences the actual state of art of BioMarin´s drugs under evaluation, including BMN-111. Has said, BioMarin predicts to have results from … Continue reading

April 20, 2014
by inesp.alves
2 Comments

Stages of implementation of BMN-111 in a short summary

BMN 111 Clinical Studies in Achondroplasia (information by: BioMarin)   1-Double-Blind, Placebo-Controlled Phase 1 Study (Protocol 111-101) Results of a two-part, double-blind, placebo-controlled Phase 1 study (Protocol 111-101) conducted in 48 healthy adult volunteers demonstrated that systemic exposure to BMN … Continue reading

September 18, 2013
by inesp.alves
2 Comments

BMN-111 going forward!!!

17 Sep 2013  BioMarin Provides Highlights Ahead of Research and Development Day BMN 111 for the Treatment of Achondroplasia   The company announced today that based on recent meetings with the FDA and the Medicines and Healthcare Products Regulatory Agency … Continue reading

May 20, 2013
by inesp.alves
0 comments

Costs and profits around orphan drugs

At the Bloomberg site, an article released on the 23rd January 2012, was written about BioMarin´s achondroplasia treatment and specially the financial involvement of that. Important article to read and to know more the inside of the rare diseases pharmaceutical profits. … Continue reading

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