Beyond Achondroplasia

Growing together with Clara

December 10, 2017
by inesp.alves
15 Comments

Update on BioMarin´s Achondroplasia Clinical Development Program

BioMarin Pharmaceutical sent to patient organizations an update of the clinical development program for BMN-111. The current status of the achondroplasia clinical programme is described in the document below. BioMarin’s investigational therapy for achondroplasia, BMN 111, is currently under investigation … Continue reading

October 20, 2016
by inesp.alves
3 Comments

BioMarin presents report on phase 2 study of Vosoritide

BioMarin presented on the 19th October 2016 a press release on the data for Vosoritide in phase 2,  during the ASHG 2016 meeting. There is no overall novelty in this latest press release (19th Oct 2016) when comparing to the previous press … Continue reading

June 30, 2016
by inesp.alves
0 comments

State of the art in achondroplasia

In the present, achondroplasia has more opportunities in the scientific search for a treatment than almost any other rare disease has. And this is very amazing. There are some relevant points in the science that is being produced for achondroplasia: … Continue reading

May 17, 2016
by inesp.alves
0 comments

Questions and answers – BMN-111 Drug Trial

LP Doc Talk is a project of Growing Stronger, a foundation based in S. Francisco, California and founded by Amer and Munira Haider, parents of Ahmin,a boy now with 7 years-old, born with achondroplasia. When I started to search about achondroplasia, I … Continue reading

April 21, 2016
by inesp.alves
6 Comments

Update in multicenter study from BioMarin

A Multicenter, Multinational Clinical Assessment Study for Pediatric Patients With Achondroplasia Eligibility Ages Eligible for Study: 2 Years to 13 Years Genders Eligible for Study: Both Accepts Healthy Volunteers: No Sampling Method: Probability Sample Study Population Approximately 200 patients will … Continue reading

October 15, 2015
by inesp.alves
0 comments

BMN-111 phase 2 results presented at the ASBMR 2015 meeting

Dr. Melita Irving, the clinical geneticist who is coordinating the phase 2 clinical trial of BMN-111 in London, presented at the American Society for Bone and Mineral Research Annual 2015 Meeting (ASBMR) the initial six-month data from the first three … Continue reading

August 4, 2015
by inesp.alves
0 comments

BMN-111 Phase 3 ancillary cohort

BioMarin press release 3rd August 2015 “A major event in the second quarter of BioMarin Pharmaceutical Inc. was the completion and results of the Phase 2 study with vosoritide (future BMN-111 commercial designation) for the treatment of achondroplasia. BioMarin related that … Continue reading

June 18, 2015
by inesp.alves
1 Comment

BMN-111 – 6 key tidbits

Comments by Mark Schoenebaum, Biotech/Pharma analyst since 2000 MD, Johns Hopkins School of Medicine, 2000 BA, Indiana University, 1995 6 key tidbits:  1)      Phase 3 could enroll 50-100 patients and could be ~1 year in duration. Patient natural history could serve … Continue reading

June 18, 2015
by inesp.alves
2 Comments

Breaking news – BMN-111 Phase 2 proof-of-concept report

Vosoritide is the name we will know from now on. It’s the generic name of BMN-111 Report from the StreetInsider.com BioMarin announced positive results of a Phase 2 proof-of-concept and dose finding study of BMN 111 (vosoritide), an analog of … Continue reading

January 20, 2015
by inesp.alves
1 Comment

BMN-111 clinical trial update

Recently, I had valid information that BioMarin announced that the measurement study is now open for recruitment again for the multicenter study. However, BioMarin didn´t change the age requirement, so children still need to be 4.5 years or older. The person … Continue reading

Translate »