Fibrodysplasia ossificans progressiva
RSS feedNew data on FOP flare-ups and the efficacy of garetosmab
According to a post-hoc analysis of the results of the Lumina-1 phase II trial of garetosmab versus placebo in fibrodysplasia ossificans progressiva (FOP) : almost three-quarters (71%) of participants in the placebo group experienced inflammatory flare-ups during the first 28 weeks of the trial, i.e. the double-blind phase; the majority (59%) developed a new heterotopic … [Read more]
Congenital hallux valgus also occurs in BMPR1B-related dysplasias
The mother of a three-month-old girl with bilateral congenital hallux valgus, informed via the internet and social networks of the possibility that her daughter had fibrodysplasia ossificans progressiva (FOP), consulted a bone disease reference centre. Pending the sequencing of the girl’s entire genome, precautions linked to the risk of FOP, such as suspending the vaccination … [Read more]
Targeting the ACVR1 gene or interleukin-1 in fibrodysplasia ossificans progressiva
Two of the therapeutic avenues explored in fibrodysplasia ossificans progressiva (FOP) have been the subject of recent publications: a report of four cases of administration of interleukin-1 inhibitors (anakinra and canakinumab) for up to six years in patients aged between 23 months and 15 years, with a significant reduction in disease flare-ups from 1.51 to … [Read more]
Overview of neuromuscular diseases
After a brief reminder of the structure of the motor unit and the various modes of inheritance, this document provides short descriptions of the neuromuscular diseases that are part of our scope at AFM-Téléthon, as well as how to manage and treat them. For each group of diseases, as well as in the motor unit … [Read more]
Garetosmab is effective in fibrodysplasia ossificans progressiva
The Phase II LUMINA 1 trial assessed the safety and efficacy of garetosmab, a monoclonal antibody that blocks activin A signaling pathways, in 44 adults with fibrodysplasia ossificans progressiva (FOP). The number and volume of new heterotopic ossifications, as well as that of soft-tissue inflammatory flare-ups, were significantly reduced on garetosmab compared with placebo. During … [Read more]
Russian success with tofacitinib in FOP
Treatment with tofacitinib 5 mg twice daily was evaluated in 13 children with chronic fibrodysplasia ossificans aged 2.2 to 19.6 years: it reduced the mean number of heterotopic ossification flare-ups from 10 in the 12 months preceding the start of treatment to 0 in the 24 months that followed. thirty-one per cent of patients showed … [Read more]
