Since the discovery of induced Pluripotent Stem cells, this kind of cell, which can become most types of cells in the organism, have been used in a variety of research programs. Now it's being used in drug discovery for achondroplasia, in this case, to test statins for their capacity of inducing differentiation of these cells into cartilage cells.

There's News on the Phase 2 clinical trial for BMN-111 from BioMarin. The company is enrolling more children into the clinical trial, which is an indication that the data for the Phase 2 is favorable and with reduced side effects.

BioMarin announced August 27, 2021, that the European Commission (EC) has granted marketing authorization for VOXZOGO® (vosoritide) for the treatment of children with achondroplasia starting at age of 2 years until the closure of growth plates, which occurs after puberty. This treatment is administered once a day by injection.

Svein Fredwall, MD, Ph.D., expert working on TRS National Resource Centre for Rare Disorders, Sunnaas Rehabilitation Hospital, in Norway, has been publishing relevant research in Skeletal Dysplasia, in particular, on the effects and impact achondroplasia has on adults. This article data is based on the ongoing project 'The Norwegian Adult Achondroplasia Study'.

The American Academy of Pediatrics (APP) Council on Genetics published an update of the clinical report "Health Supervision for Children With Achondroplasia" by Trotter T & Hall J, 2005, on the June 2020 issue of Pediatrics. This clinical report offers age group-specific guidance, including information on counseling expectant parents.  

Inês Alves, Beyond Achondroplasia and ANDO Portugal founder, is representing the patients and contributing to the rare bone disease section of the 9th International Conference on Children's Bone Health as a member of a working group who organized this event by organizing a pre-meeting workshop.