Beyond Achondroplasia

Growing together with Clara

January 7, 2015
by inesp.alves
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Challenges of treating growth plate in children with achondroplasia

In “Achondroplasia: A view to the future options emerging from the benchside”, Narayana, J., Horton, W.A, 2013, there is a very interesting point that reflects the challenges of bringing FGFR3-based therapies to the bedside. “Despite the considerable recent progress in the … Continue reading

November 20, 2014
by inesp.alves
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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

October 17, 2014
by inesp.alves
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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

July 4, 2014
by inesp.alves
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A summary of achondroplasia´s potential treatments

I have been following for several months the search work of prof. Morrys Kaisermann around achondroplasia latest researchs. He keeps a blog about achondroplasia named “Acondroplasia-Achondroplasia” and his lastest post is a brilliant synopsis of the current achondroplasia potential treatments and … Continue reading

June 17, 2014
by inesp.alves
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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
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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

March 31, 2014
by inesp.alves
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BMN-111 after 30 years of The Orphan drug Act

There is still some years ahead until the BioMarin´s study and clinical trial with BMN-111 to reach the estimated study completion date: 1) A Multicenter, Multinational Clinical Assessment Study for Pediatric Patients With Achondroplasia 2) A Phase 2 Study of BMN 111 … Continue reading

February 12, 2014
by inesp.alves
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A Phase 2 Study of BMN 111 to Evaluate Safety, Tolerability, and Efficacy in Children With Achondroplasia (ACH)

BioMarin activated now the Phase 2 study in the Clinicaltrials.gov. Another step forward! Only Chicago hospital is recruiting for now.  BMN 111 is being administered by a subcutaneous Injection and as a morning dose in one of the following daily dosing regimens: … Continue reading

January 14, 2014
by inesp.alves
4 Comments

BMN-111 phase 2 – the first patient

January 14, 2014 Press release BioMarin Doses First Patient in Phase 2 Trial With BMN 111 for the Treatment of Children With Achondroplasia BioMarin Pharmaceutical announced today that it has dosed the first child in the Phase 2 trial with … Continue reading

December 2, 2013
by inesp.alves
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Vessel dilator peptide – achondroplasia future treatment?

Vessel Dilator and C-Type Natriuretic Peptide Enhance the Proliferation of Human Osteoblasts Anne Lenz, Meghan Bennett, William P Skelton and David L Vesely (2010) C-natriuretic peptide (CNP) has been shown to regulate proliferation of mouse and rat osteoblasts. Genetic deletion of CNP results in dwarfism. … Continue reading

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