Pat Furlong: search on Z-Library

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1

What compassionate use means for gene therapies

Chapman, Carolyn Riley, Moch, Kenneth I., McFadyen, Andrew, Kearns, Lisa, Watson, Tom, Furlong, Pat, Bateman-House, Alison

Journal:

Nature Biotechnology

Year:

2019

Language:

english

File:

PDF, 735 KB

english, 2019

2

Recommendations for the development of rare disease drugs using the accelerated approval pathway and for qualifying biomarkers as primary endpoints

Kakkis, Emil D, O’Donovan, Mary, Cox, Gerald, Hayes, Mark, Goodsaid, Federico, Tandon, PK, Furlong, Pat, Boynton, Susan, Bozic, Mladen, Orfali, May, Thornton, Mark

Journal:

Orphanet Journal of Rare Diseases

Year:

2015

Language:

english

File:

PDF, 628 KB

english, 2015

3

How a patient advocacy group developed the first proposed draft guidance document for industry for submission to the U.S. Food and Drug Administration

Furlong, Pat, Bridges, John F. P., Charnas, Lawrence, Fallon, Justin R., Fischer, Ryan, Flanigan, Kevin M., Franson, Timothy R., Gulati, Neera, McDonald, Craig, Peay, Holly, Sweeney, H. Lee

Journal:

Orphanet Journal of Rare Diseases

Year:

2015

Language:

english

File:

PDF, 553 KB

english, 2015

4

Improving Access and Quality of Health Care in the United States: Shared Goals Among Patient Advocates

Oehrlein, Elisabeth M., Harris, Jason, Balch, Alan, Furlong, Pat, Hargis, Eric, Woolley, Mary, Perfetto, Eleanor

Journal:

The Patient: Patient-Centered Outcomes Research

Year:

2020

File:

PDF, 510 KB

2020

5

Design issues in the measurement of quality of life in a trial of two prenatal diagnostic technologies: Genetic amniocentesis and chorionic villi sampling

Marie Townsend, David Feeny, Bill Furlong, Darrell Tomkins, Gail Robinson, Pat Mohide, George Torrance

Journal:

Controlled Clinical Trials

Year:

1990

Language:

english

File:

PDF, 128 KB

english, 1990

6

Letter 2

Filippo Buccella, Pat Furlong, Sally Hofmeister, Elizabeth Vroom

Journal:

Neuromuscular Disorders

Year:

2010

Language:

english

File:

PDF, 115 KB

english, 2010

7

Response

Annemieke Aartsma-Rus, Pat Furlong, Elizabeth Vroom, Gert-Jan van Ommen, Erik Niks, Chiara Straathof, Jan Verschuuren, Annemieke Aartsma-Rus, Alessandra Ferlini, Lynn Hagger, Emma Heslop, Veronika Kar

Journal:

Neuromuscular Disorders

Year:

2011

Language:

english

File:

PDF, 44 KB

english, 2011

8

The risks of therapeutic misconception and individual patient (n = 1) “trials” in rare diseases such as Duchenne dystrophy

Annemieke Aartsma-Rus, Pat Furlong, Elizabeth Vroom, Gert-Jan van Ommen, Erik Niks, Chiara Straathof, Jan Verschuuren, Annemieke Aartsma-Rus, Alessandra Ferlini, Lynn Hagger, Emma Heslop, Veronika Kar

Journal:

Neuromuscular Disorders

Year:

2011

Language:

english

File:

PDF, 91 KB

english, 2011

9

Trust It or Trash It? A Tool for Evaluating the Quality of Genetic Information

McInerney, Joseph D., Boguski, Mark S., Furlong, Pat, Heywood, Benjamin

Journal:

Genetic Testing and Molecular Biomarkers

Year:

2010

Language:

english

File:

PDF, 130 KB

english, 2010

10

Measuring clinical effectiveness of medicinal products for the treatment of Duchenne muscular dystrophy

Lynn, Stephen, Aartsma-Rus, Annemieke, Bushby, Kate, Furlong, Pat, Goemans, Nathalie, De Luca, Annamaria, Mayhew, Anna, McDonald, Craig, Mercuri, Eugenio, Muntoni, Francesco, Pohlschmidt, Marita, Vers

Journal:

Neuromuscular Disorders

Year:

2015

Language:

english

File:

PDF, 1.04 MB

english, 2015

11

Large-scale serum protein biomarker discovery in Duchenne muscular dystrophy

Hathout, Yetrib, Brody, Edward, Clemens, Paula R., Cripe, Linda, DeLisle, Robert Kirk, Furlong, Pat, Gordish-Dressman, Heather, Hache, Lauren, Henricson, Erik, Hoffman, Eric P., Kobayashi, Yvonne Moni

Journal:

Proceedings of the National Academy of Sciences

Year:

2015

Language:

english

File:

PDF, 851 KB

english, 2015

12

Mothers’ psychological adaptation to Duchenne/Becker muscular dystrophy

Peay, Holly L, Meiser, Bettina, Kinnett, Kathleen, Furlong, Pat, Porter, Kathryn, Tibben, Aad

Journal:

European Journal of Human Genetics

Year:

2015

Language:

english

File:

PDF, 264 KB

english, 2015

13

1St workshop on upper-extremity assistive technology for people with duchenne: state of the art, emerging avenues, and challenges

Bergsma, Arjen, Lobo-Prat, Joan, Vroom, Elizabeth, Furlong, Pat, Herder, Just L.

Journal:

Neuromuscular Disorders

Year:

2016

Language:

english

File:

PDF, 942 KB

english, 2016

14

Complex mixtures, complex responses: Assessing pharmaceutical mixtures using field and laboratory approaches

Schoenfuss, Heiko L., Furlong, Edward T., Phillips, Pat J., Scott, Tia-Marie, Kolpin, Dana W., Cetkovic-Cvrlje, Marina, Lesteberg, Kelsey E., Rearick, Daniel C.

Journal:

Environmental Toxicology and Chemistry

Year:

2016

Language:

english

File:

PDF, 1.86 MB

english, 2016

15

How close are we to a cure for Duchenne muscular dystrophy?

Journal:

Expert Opinion on Orphan Drugs

Year:

2014

Language:

english

File:

PDF, 447 KB

english, 2014