Plain Language Summaries

Standalone, citable summaries published in the Future Science Group journals written to be understood by a lay audience

Rare Diseases

Plain language summary of the design of the APPEAR-C3G study which showed how iptacopan works in people with complement 3 glomerulopathy

This Plain Language Summary of Publication from Future Rare Diseases describes the results from an ongoing study which looks at whether a new medicine called iptacopan can be used to treat a rare kidney disease called complement 3 glomerulopathy (C3G).

Read the full article here.

The original article on which this Plain Language Summary of Publication is based is titled ‘Alternative Complement Pathway Inhibition With Iptacopan for the Treatment of C3 Glomerulopathy-Study Design of the APPEAR-C3G Trial’ and was published in Kidney International Reports. Read the original article here.

The effects of palovarotene in patients with fibrodysplasia ossificans progressiva: a plain language summary

This plain language summary from Future Rare Diseases describes the results of the MOVE study, which investigated the drug palovarotene in the treatment of patients with fibrodysplasia ossificans progressiva (FOP).

Read the full article here.

The original article on which this plain language summary is based is titled ‘Reduction of New Heterotopic Ossification (HO) in the Open-Label, Phase 3 MOVE Trial of Palovarotene for Fibrodysplasia Ossificans Progressiva (FOP)’ and was published in the Journal of Bone and Mineral Research. Read the original article here.

Plain language summary of a study looking at the age at diagnosis and time to start of treatment in individuals with mucopolysaccharidosis type I (MPS I)

This plain language summary looks at a study which investigated the age when individuals were diagnosed with attenuated or severe Mucopolysaccharidosis type I (also called MPS I) and the time taken for them to start treatment.

Read the full article here.

mucopolysaccharidosis type I

You can read the full article called ‘Improvement in time to treatment, but not time to diagnosis, in patients with mucopolysaccharidosis type I’ published in the journal Archives of Disease in Childhood for free at: https://adc.bmj.com/content/106/7/674.long.

The impact of fibrodysplasia ossificans progressiva (FOP) on patients and their family members: results from an international burden of illness survey

The first international fibrodysplasia ossificans progressiva burden of illness survey was carried out to learn more about the physical impact, quality of life impact, and economic impact of the condition on people with FOP and their families. This new plain language summary in Future Rare Diseases discusses the findings from this survey.

Read the full article here.

The original article on which this summary is based is called ‘The Impact of Fibrodysplasia Ossificans Progressiva (FOP) on Patients and their Family Members: Results from an International Burden of Illness Survey’ and was originally published in the journal Expert Review of Pharmacoeconomics & Outcomes Research. The original article can be read here.

Plain language summary of a study looking at the age at diagnosis and time to start of treatment in individuals with mucopolysaccharidosis type I (MPS I)

This new plain language summary in Future Rare Diseases discusses a study using data from the MPS I Registry that looks at the age when individuals were diagnosed with MPS I and the time taken for them to start treatment with enzyme replacement therapy or hematopoietic stem cell transplantation.

Read the full article here.

The original article on which this summary is based is called ‘Improvement in time to treatment, but not time to diagnosis, in patients with mucopolysaccharidosis type I’ and was originally published in the journal Archives of Disease in Childhood. The original article can be read here.