Beyond Achondroplasia

Growing together with Clara

June 30, 2018
by inesp.alves
3 Comments

Vosoritide is now in study in children under 5 years old

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

5th Nordic Skeletal Dysplasia Symposium – full session dedicated to achondroplasia

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

The TransCon CNP for achondroplasia – Ascendis Pharma publications

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

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
6 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
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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
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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

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