RareWays is a research directory. It helps you understand the science. It is not medical advice. Always discuss your care options with your healthcare team.
Coverage: 2020-01-01 onwards, with up to 300 results per query from PubMed and Europe PMC and separate limits on other sources. This is a selected index, not a complete literature search.
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Hypophosphatasia: Research Summary
Printed from RareWays (rareways.org)
For general awareness only. Not medical advice. Discuss all care options with your healthcare team.
5 Most Recent Research Articles
- 1.
Insurance and altruism values of a novel treatment for hypophosphatasia: a stated preference survey in the United States
Andrew J. Messali et al., Figshare (15 October 2026)
https://doi.org/10.6084/m9.figshare.34530456
- 2.
Correction to: Hypophosphatasia: low penetrance of pathogenic and likely-pathogenic ALPL variants identified through an unselected biorepository.
, Journal of bone and mineral research : the official journal of the American Society for Bone and Mineral Research (11 October 2026)
https://pubmed.ncbi.nlm.nih.gov/42859419/
- 3.
Clinical and molecular characterization of an Egyptian cohort with autosomal recessive hypophosphatasia
Dina El Dessouki et al., Scientific Reports (8 October 2026)
https://doi.org/10.1038/s41598-026-71686-w
- 4.
Prevalence of hypophosphatasia in general and selected patient populations: a systematic review.
Schäfer Valentin S et al., Osteoporosis international : a journal established as result of cooperation between the European Foundation for Osteoporosis and the National Osteoporosis Foundation of the USA (7 October 2026)
https://pubmed.ncbi.nlm.nih.gov/42844428/
- 5.
Burden of disease in US patients with skeletal versus muscular or pain manifestations of hypophosphatasia: An analysis from the Global HPP Registry.
Kishnani Priya S et al., Bone (1 October 2026)
https://pubmed.ncbi.nlm.nih.gov/42365936/
Clinical Trials : Australian Sites
Ask your doctor whether you or your child may be eligible for any of these trials. Each has its ID and full eligibility criteria at the link shown.
- 1.
Phase 3 Study of ALXN1850 in Treatment-Naïve Pediatric Participants With HPP
NCT06079359 · Active (not recruiting) · Phase 3 · Alexion Pharmaceuticals, Inc.
Ages 2 to 11
https://clinicaltrials.gov/study/NCT06079359
- 2.
Phase 3 Study of ALXN1850 in Pediatric Participants With HPP Previously Treated With Asfotase Alfa
NCT06079372 · Active (not recruiting) · Phase 3 · Alexion Pharmaceuticals, Inc.
Ages 2 to 11
https://clinicaltrials.gov/study/NCT06079372
- 3.
Phase 3 Study of ALXN1850 Versus Placebo in Adolescent and Adult Participants With HPP Who Have Not Previously Been Treated With Asfotase Alfa
NCT06079281 · Active (not recruiting) · Phase 3 · Alexion Pharmaceuticals, Inc.
Ages 12 to 130
https://clinicaltrials.gov/study/NCT06079281
- 4.
Open-Label Study of Asfotase Alfa in Infants and Children ≤ 5 Years of Age With Hypophosphatasia (HPP)
NCT01176266 · Completed · Phase 2 / Phase 3 · Alexion Pharmaceuticals, Inc.
Ages 1 Minute to 5
https://clinicaltrials.gov/study/NCT01176266
Source: RareWays research directory. Data from PubMed, Europe PMC, OpenAlex, ClinicalTrials.gov.
Always verify information with your healthcare team before making any decisions about your care.
Hypophosphatasia
Hypophosphatasia is an inherited condition caused by changes in the ALPL gene, which lower an enzyme the body needs to harden bones and teeth. Severity ranges from life-threatening disease in newborns to early loss of baby teeth, fractures and bone pain in adults. Enzyme replacement therapy (asfotase alfa) is available for some patients.
This overview is general information, not an individual medical assessment. Research is selected automatically and may include mismatches. How we select and explain research.
Most Recent Research
To estimate the insurance and altruism values of a novel hypothetical treatment on survival gains for perinatal-/infantile-onset hypophosphatasia (HPP), a rare, severe, inherited disease, which—if untreated—leads to early mortality in children. Residents of the United States aged 21–54 years from the general population participated in a web-based, cross-sectional, prospective, stated preference survey. The insurance value module included respondents planning to have children, whereas the altruism value module included respondents not planning to have children in the future. A multiple random staircase survey design was utilized to assess respondents’ willingness to pay (WTP) for insurance coverage of the novel HPP treatment offering survival gains. Among the 511 eligible respondents, 50.3% (n = 257) and 49.7% (n = 254) were included in the insurance value and altruism value modules, respectively. Overall, 72.2% of the respondents were aged 30–49 years (insurance module: 72.4% and altruism module: 72.0%) and 52.8% were men (insurance module: 56.8% and altruism module: 48.8%). For generous coverage of the novel HPP treatment, the mean WTP was $10.35/month or $124.22/year as additional health insurance premiums for the insurance value module (insurance value makes 90.1% of the total treatment value). The mean WTP was $9.40/month or $112.85/year as taxes for the altruism value module. The high insurance and altruism values for the novel HPP treatment, which can significantly improve survival, suggest that health gains for children may be valued more highly than similar health gains for adults, especially in a rare disease context. Hypophosphatasia (HPP) is a rare, genetic disease and can be life-threatening in those who have first symptoms at less than 6 months of age. This study assessed insurance and altruism values of a hypothetical novel treatment for children with HPP diagnosed within the first 6 months of life in the United States (US). Insurance value was defined as the value (individuals’ willingness to pay for an insurance policy that covers the treatment) placed on the insurance coverage of the new treatment among adults (aged 21–54 years) in the US general population planning to have children (or more children among those already with children) in the future. Insurance value group accounts for future HPP diagnosis in their children. Altruism value group was defined as the value placed on the new treatment among adults not planning to have one or more children in the future. Among 511 participants included, 257 were in the insurance group and 254 were in the altruism group. Insurance value group participants were willing to pay a monthly premium of $10.35 or $124.22 yearly to secure access to the novel treatment for the future generation. Altruism value group participants were willing to pay a monthly government tax of $9.40 or $112.85 yearly so that other children in the society could benefit from the novel treatment. These findings suggested that US adults were willing to pay additional health insurance premium or tax to have access to a treatment, which improves survival of the next generations in case of HPP diagnosis.
Common Questions
What is Hypophosphatasia?
Hypophosphatasia is an inherited condition caused by changes in the ALPL gene, which lower an enzyme the body needs to harden bones and teeth. Severity ranges from life-threatening disease in newborns to early loss of baby teeth, fractures and bone pain in adults. Enzyme replacement therapy (asfotase alfa) is available for some patients.
How many clinical trials are available for Hypophosphatasia?
RareWays currently indexes 21 clinical trials for Hypophosphatasia, of which 1 is actively recruiting. These counts cover indexed trials across countries, not just your country. An Australian site does not necessarily mean that site is recruiting. Check locations, eligibility and current availability with the study team.
Where does the research data for Hypophosphatasia come from?
RareWays aggregates research from PubMed, Europe PMC, OpenAlex, and ClinicalTrials.gov. Data is refreshed automatically every day. All articles and trials link directly to their original sources.
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