The articles provide a step-by-step discussion about scientific aspects of achondroplasia. We review the mutation origin and the molecular mechanisms of achondroplasia. However, most of the articles intend to share information about potential therapeutic strategies for achondroplasia.
The blog started as a personal collaboration initiative with Growing Stronger, the non profit, parents’ organization devoted to stimulate and fund research on pharmacological therapies for achondroplasia.
30/04/2018.Treating achondroplasia: a review of the therapy landscape with focus on statins
14/03/2018.Treating Achondroplasia: frequent questions about treatment
01/02/2018.Treating achondroplasia: sixth year online and news
11/01/2018.Treating achondroplasia: TransCon CNP showed positive results in pre-clinical tests
25/10/2017.Treating achondroplasia: thirty months with vosoritide
07/08/2017.Treating achondroplasia: update on the current potential therapies
11/01/2017. Treating Achondroplasia: fifth year online
12/12/2016.Treating Achondroplasia: Phase 3 study with vosoritide started in Australia
19/10/2016. Treating Achondroplasia: results from the fourth cohort of the phase 2 study with vosoritide have been released
18/10/2016. Treating Achondroplasia: highlights from the recent literature: Fluazuron and CNP
24/09/2016. Vosoritide one year phase 2 results of the third cohort to be presented in the next ASHG 2016 meeting
17/05/2016.Treating achondroplasia: NVP-BGJ398, a tyrosine kinase inhibitor, restores bone growth in a model of achondroplasia
21/04/2016. Treating achondroplasia: news about the clinical development of vosoritide
10/03/2016. Stopping FGFR3 production to treat achondroplasia (part 1)
29/02/2016. The Rare Disease Day 2016
11/01/2016. Treating Achondroplasia: fourth year online
05/01/2016. FGFR4: a problem for ligand trap therapeutic strategies in achondroplasia?
06/11/2015. Treating achondroplasia: the role of Tyrosine Kinase Inhibitors
27/09/2015. Treating achondroplasia: B-701, a new antibody anti-FGFR3 and more news
03/08/2015. Biomarin announces first patient in the fourth cohort of the phase 2 study
30/07/2015. Treating achondroplasia: news from the vosoritide clinical development program
05/07/2015. Treating achondroplasia: meclizine has positive effects for the foramen magnum in a model of achondroplasia
17/06/2015. Vosoritide (BMN-111) induces 50% increase in growth velocity in children with achondroplasia
28/05/2015. Treating achondroplasia: BMN-111 and the catch up growth phenomenon
17/05/2015. Treating achondroplasia: news about BMN-111
30/03/2015. Treating achondroplasia: the history of the development of BMN-111
14/03/2015. Treating achondroplasia: just one's right
31/01/2015. Biomarin updates the information about the BMN-111 Phase 2 study in clinicaltrials.gov
20/01/2015. The Treating Achondroplasia Blog: Third Year Online
15/12/2014. Treating achondroplasia with meclizine / meclozine
01/12/2014. Treating achondroplasia with statins
20/08/2014. FGFR3 and growth plate literature news
18/06/2014. Treating achondroplasia: where we are now
31/05/2014. FGF18, a key player for bone growth
19/05/2014. News from Biomarin about BMN-111
07/03/2014. Treating achondroplasia: NC-2, a new CNP analogue
10/02/2014. Biomarin includes the BMN-111 phase 2 study at the ClinicalTrials.gov
03/02/2014. Growing Stronger and LPA to receive proposals for funding research on skeletal dysplasias
27/01/2014. Blocking the MAPK cascade to treat achondroplasia
20/01/2014. Why do chondrocytes stop in achondroplasia? part 2
14/01/2014. Biomarin announced that the Phase 2 study of BMN-111, the first potential pharmacological therapy for achondroplasia, has just started
08/01/2014. Treating achondroplasia: second year online
25/12/2013. Why do chondrocytes stop in achondroplasia?
11/12/2013. Meclizine / meclozine for achondroplasia?
30/11/2013. Natriuretic peptides for achondroplasia: what's new?
02/11/2013. The First International Dwarfism Congress of Portugal
19/10/2013. Learning about signaling cascades
12/10/2013. New evidence of CNP signaling cascade relevance in bone growth
20/09/2013. A new ligand trap strategy to treat achondroplasia
17/09/2013. BMN-111 phase 2 study has been authorized by the FDA
14/09/2013. On air, the Brazilian reference site for bone dysplasias
10/09/2013. More news about BMN-111
08/09/2013. Repurposing of old drugs: is there an opportunity for achondroplasia?
07/09/2013. BMN-111 in The International Skeletal Disease Society - ISDS 2013 Meeting
01/09/2013. Stem cell therapy for achondroplasia?
25/07/2013. BMN-111 news from July 25th Biomarin conference call
08/07/2013. The Blog has now a Glossary Page
16/06/2013. Interfering in the production of FGFR3: a potential strategy to treat achondroplasia
28/05/2013. Exploring the use of aptamers to treat achondroplasia
09/05/2013. Researchers identify a new class of FGFR inhibitors
04/05/2013. Biomarin updated information about the clinical development study of children with achondroplasia
26/04/2013. The mechanism of action of CNP in the FGFR3 pathway showed by a new study
30/03/2013. Capturing fibroblast growth factors to treat cancer. Could this strategy be used to treat achondroplasia?
05/02/2013. Treating achondroplasia with anti-FGFR3 specific peptides
30/01/2013. The Acondroplasia - Achondroplasia blog: first year online
26/01/2013. The Rare Disease Day 2013
19/01/2013. Developing medicines for rare conditions
04/01/2013.Treating achondroplasia: the great challenge ahead
01/01/2013. Treating achondroplasia: concepts and misconcepts about the potential therapies in development
14/12/2012. Achondroplasia and the new potential clinical therapies: who can benefit from them?
09/12/2012. A new phase 1 clinical trial of an anti-FGFR3 antibody in Multiple Myeloma. Could this antibody be used in achondroplasia?
03/11/2012. Enzymes of the FGFR3 cascade may be appropriate targets for the treatment of achondroplasia
12/10/2012. A newly identified peptide blocks FGFR3 and rescue growth in a tanatophoric dysplasia mouse model
10/10/2012. MK-4 reduces the production of FGFR3 in liver cancer cells. A new pharmacological approach for achondroplasia?
27/08/2012. Parathyroid Hormone to treat achondroplasia? Part 2: medical and experimental use of PTH
30/06/2012. Challenges for the treatment for achondroplasia: to bodly go where no man has gone before
13/06/2012. Challenges for the treatment for achondroplasia: from drug development to drug delivery, part 2
28/04/2012. Challenges for the treatment for achondroplasia: from drug development to drug delivery, part 1
19/03/2012. The last stronghold: the growth plate cartilage
19/02/2012. The road to manage achondroplasia
19/02/2012. Understanding achondroplasia
12/01/2012. A quick introduction