Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation

Search Page

Filters

My Custom Filters

Publication date

Text availability

Article attribute

Article type

Additional filters

Article Language

Species

Sex

Age

Other

Search Results

4,458 results

Filters applied: . Clear all
Results are displayed in a computed author sort order. The Publication Date timeline is not available.
Page 1
Investigating health-related quality of life in rare diseases: a case study in utility value determination for patients with CLN2 disease (neuronal ceroid lipofuscinosis type 2).
Gissen P, Specchio N, Olaye A, Jain M, Butt T, Ghosh W, Ruban-Fell B, Griffiths A, Camp C, Sisic Z, Schwering C, Wibbeler E, Trivisano M, Lee L, Nickel M, Mortensen A, Schulz A. Gissen P, et al. Among authors: jain m. Orphanet J Rare Dis. 2021 May 12;16(1):217. doi: 10.1186/s13023-021-01829-x. Orphanet J Rare Dis. 2021. PMID: 33980287 Free PMC article.
Evaluating discrete choice experiment willingness to pay [DCE-WTP] analysis and relative social willingness to pay [RS-WTP] analysis in a health technology assessment of a treatment for an ultra-rare childhood disease [CLN2].
Moro D, Schlander M, Telser H, Sola-Morales O, Clark MD, Olaye A, Camp C, Jain M, Butt T, Bakshi S. Moro D, et al. Among authors: jain m. Expert Rev Pharmacoecon Outcomes Res. 2022 Jun;22(4):581-598. doi: 10.1080/14737167.2022.2014324. Epub 2022 Feb 23. Expert Rev Pharmacoecon Outcomes Res. 2022. PMID: 34877915 Free article.
Exploring concurrent validity of the CLN2 Clinical Rating Scale: Comparison to PedsQL using cerliponase alfa clinical trial data.
Specchio N, Gissen P, de Los Reyes E, Olaye A, Camp C, Curteis T, Griffiths A, Butt T, Cohen-Pfeffer J, Slasor P, Sisic Z, Jain M, Schulz A. Specchio N, et al. Among authors: jain m. PLoS One. 2024 May 22;19(5):e0302382. doi: 10.1371/journal.pone.0302382. eCollection 2024. PLoS One. 2024. PMID: 38776275 Free PMC article. Clinical Trial.
Clinical burden of illness in patients with phenylketonuria (PKU) and associated comorbidities - a retrospective study of German health insurance claims data.
Trefz KF, Muntau AC, Kohlscheen KM, Altevers J, Jacob C, Braun S, Greiner W, Jha A, Jain M, Alvarez I, Lane P, Schröder C, Rutsch F. Trefz KF, et al. Among authors: jain m. Orphanet J Rare Dis. 2019 Jul 22;14(1):181. doi: 10.1186/s13023-019-1153-y. Orphanet J Rare Dis. 2019. PMID: 31331350 Free PMC article.
4,458 results