Works matching IS 15524868 AND DT 2023 AND VI 193 AND IP 1

Results: 14

Cover Image, Volume 193, Number 1, March 2023.

Published in:: American Journal of Medical Genetics. Part C: Seminars in Medical Genetics, 2023, v. 193, n. 1, p. i, doi. 10.1002/ajmg.c.31974
Publication type:: Article

Note from the editors.

Published in:

American Journal of Medical Genetics. Part C: Seminars in Medical Genetics, 2023, v. 193, n. 1, p. 5, doi. 10.1002/ajmg.c.32039

By:

Urv, Tiina K.;
Parisi, Melissa A.

Publication type:

Article

A review of economic issues for gene‐targeted therapies: Value, affordability, and access.

Published in:

American Journal of Medical Genetics. Part C: Seminars in Medical Genetics, 2023, v. 193, n. 1, p. 64, doi. 10.1002/ajmg.c.32037

By:

Garrison, Louis P.;
Lo, Andrew W.;
Finkel, Richard S.;
Deverka, Patricia A.

Publication type:

Article

When is the best time to screen and evaluate for treatable genetic disorders?: A lifespan perspective.

Published in:

American Journal of Medical Genetics. Part C: Seminars in Medical Genetics, 2023, v. 193, n. 1, p. 44, doi. 10.1002/ajmg.c.32036

By:

Parisi, Melissa A.;
Caggana, Michele;
Cohen, Jennifer L.;
Gold, Nina B.;
Morris, Jill A.;
Orsini, Joseph J.;
Urv, Tiina K.;
Wasserstein, Melissa P.

Publication type:

Article

Gene‐targeted therapies: Overview and implications.

Published in:

American Journal of Medical Genetics. Part C: Seminars in Medical Genetics, 2023, v. 193, n. 1, p. 13, doi. 10.1002/ajmg.c.32033

By:

Brooks, P J;
Urv, Tiina K.;
Parisi, Melissa A.

Publication type:

Article

Gene‐targeted therapies: Towards equitable development, diagnosis, and access.

Published in:

American Journal of Medical Genetics. Part C: Seminars in Medical Genetics, 2023, v. 193, n. 1, p. 56, doi. 10.1002/ajmg.c.32032

By:

Gaviglio, Amy M.;
Skinner, Mark W.;
Lou, Lily J.;
Finkel, Richard S.;
Augustine, Erika F.;
Goldenberg, Aaron J.

Publication type:

Article

Moving away from one disease at a time: Screening, trial design, and regulatory implications of novel platform technologies.

Published in:

American Journal of Medical Genetics. Part C: Seminars in Medical Genetics, 2023, v. 193, n. 1, p. 30, doi. 10.1002/ajmg.c.32031

By:

Lekstrom‐Himes, Julie;
Brooks, P J;
Koeberl, Dwight D.;
Brower, Amy;
Goldenberg, Aaron;
Green, Robert C.;
Morris, Jill A.;
Orsini, Joseph J.;
Yu, Timothy W.;
Augustine, Erika F.

Publication type:

Article

Are we prepared to deliver gene‐targeted therapies for rare diseases?

Published in:

American Journal of Medical Genetics. Part C: Seminars in Medical Genetics, 2023, v. 193, n. 1, p. 7, doi. 10.1002/ajmg.c.32029

By:

Yu, Timothy W.;
Kingsmore, Stephen F.;
Green, Robert C.;
MacKenzie, Tippi;
Wasserstein, Melissa;
Caggana, Michele;
Gold, Nina B.;
Kennedy, Annie;
Kishnani, Priya S.;
Might, Matthew;
Brooks, Phillip J.;
Morris, Jill A.;
Parisi, Melissa A.;
Urv, Tiina K.

Publication type:

Article

Data sharing to advance gene‐targeted therapies in rare diseases.

Published in:

American Journal of Medical Genetics. Part C: Seminars in Medical Genetics, 2023, v. 193, n. 1, p. 87, doi. 10.1002/ajmg.c.32028

By:

Lekstrom‐Himes, Julie;
Augustine, Erika F.;
Brower, Amy;
Defay, Thomas;
Finkel, Richard S.;
McGuire, Amy L.;
Skinner, Mark W.;
Yu, Timothy W.

Publication type:

Article

Whole‐genome sequencing holds the key to the success of gene‐targeted therapies.

Published in:

American Journal of Medical Genetics. Part C: Seminars in Medical Genetics, 2023, v. 193, n. 1, p. 19, doi. 10.1002/ajmg.c.32017

By:

Vockley, Jerry;
Aartsma‐Rus, Annemieke;
Cohen, Jennifer L.;
Cowsert, Lex M.;
Howell, R. Rodney;
Yu, Timothy W.;
Wasserstein, Melissa P.;
Defay, Thomas

Publication type:

Article

Scaling genetic resources: New paradigms for diagnosis and treatment of rare genetic disease.

Published in:

American Journal of Medical Genetics. Part C: Seminars in Medical Genetics, 2023, v. 193, n. 1, p. 77, doi. 10.1002/ajmg.c.32016

By:

Vockley, Jerry;
Defay, Thomas;
Goldenberg, Aaron J.;
Gaviglio, Amy M.

Publication type:

Article

Cover Image, Volume 193, Number 1, March 2023.

Published in:: American Journal of Medical Genetics. Part C: Seminars in Medical Genetics, 2023, v. 193, n. 1, p. i, doi. 10.1002/ajmg.c.31974
Publication type:: Article

Publication schedule for 2023.

Published in:: American Journal of Medical Genetics. Part C: Seminars in Medical Genetics, 2023, v. 193, n. 1, p. 3, doi. 10.1002/ajmg.c.31975
Publication type:: Article

Table of Contents, Volume 193, Number 1, March 2023.

Published in:: American Journal of Medical Genetics. Part C: Seminars in Medical Genetics, 2023, v. 193, n. 1, p. 1, doi. 10.1002/ajmg.c.31974
Publication type:: Article