June 30, 2018
by inesp.alves
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BioMarin announced on the 14th June 2018, the first administration of BMN 111 to young children with achondroplasia under 5 years old. (1) This is the BMN 111-206 study, a phase 2 study in Infants and Toddlers. What … Continue reading
March 4, 2018
by inesp.alves
3 Comments
The 5th Nordic Skeletal Dysplasia Symposium will be held in Copenhagen, on March 8-9, 2018. As stated in the Welcome note of this symposium “Skeletal dysplasias are rare diseases and the annual symposium is important to share medical knowledge with … Continue reading
December 31, 2017
by inesp.alves
3 Comments
Ascendis Pharma, a company funded in 2007, applies the TransCon technology which combines a prodrug with a sustained-release technology. This way, the company can offer products with a predictable and sustained release of an unmodified parent drug. Before heading for the … Continue reading
June 3, 2017
by inesp.alves
1 Comment
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
6 Comments
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
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
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
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
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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
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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