Works matching IS 26330040 AND DT 2022 AND VI 3

Results: 16

Building cross-border collaborations to increase diversity and accelerate rare disease drug development -- meeting report from the inaugural IndoUSrare Annual Conference 2021.

Published in:

Therapeutic Advances in Rare Disease, 2022, v. 3, p. 1, doi. 10.1177/26330040221133124

By:

Khera, Harvinder Kour;
Venugopal, Nisha;
Karur, Ramya T.;
Mishra, Rakesh;
Kartha, Reena V.;
Rajasimha, Harsha K.

Publication type:

Article

Telehealth for patients with rare epilepsies.

Published in:

Therapeutic Advances in Rare Disease, 2022, v. 3, p. 1, doi. 10.1177/26330040221076861

By:

Kramer, Zachary J.;
Brandt, Carrin;
Havens, Kathryn;
Pasupuleti, Archana;
Gaillard, William D.;
Schreiber, John M.

Publication type:

Article

Quality of life in Barth syndrome.

Published in:

Therapeutic Advances in Rare Disease, 2022, v. 3, p. 1, doi. 10.1177/26330040221093743

By:

Kim, Alexander Y.;
Vernon, Hilary;
Manuel, Ryan;
Almuqbil, Mohammed;
Hornby, Brittany

Publication type:

Article

Therapeutic angiogenesis in Buerger's disease: reviewing the treatment landscape.

Published in:

Therapeutic Advances in Rare Disease, 2022, v. 3, p. 1, doi. 10.1177/26330040211070295

By:

Ribieras, Antoine J.;
Ortiz, Yulexi Y.;
Zhao-Jun Liu;
Velazquez, Omaida C.

Publication type:

Article

End-stage crystalline maculopathy with retinal atrophy in Sjögren-Larsson syndrome: a case report and review of the literature.

Published in:

Therapeutic Advances in Rare Disease, 2022, v. 3, p. 1, doi. 10.1177/26330040221122496

By:

Lambert, Lester H.;
Shaikh, Noreen;
Marx, Jeffrey L.;
Ramsey, David J.

Publication type:

Article

Racial differences in the dermatological manifestations of tuberous sclerosis complex and the potential effects on diagnosis and care.

Published in:

Therapeutic Advances in Rare Disease, 2022, v. 3, p. 1, doi. 10.1177/26330040221140125

By:

Pounders, Ashley J.;
Rushing, Gabrielle V.;
Mahida, Sonal;
Sanny Nonyane, Bareng Aletta;
Thomas, Emily A.;
Tameez, Rabiah Sundus;
Gipson, Tanjala T.

Publication type:

Article

What are the benefits of the anti-FGF23 antibody burosumab on the manifestations of X-linked hypophosphatemia in adults in comparison with conventional therapy? A review.

Published in:

Therapeutic Advances in Rare Disease, 2022, v. 3, p. 1, doi. 10.1177/26330040221074702

By:

Lafage-Proust, Marie-Hélène

Publication type:

Article

Clinical evidence of interventions assessed in Friedreich ataxia: a systematic review.

Published in:

Therapeutic Advances in Rare Disease, 2022, v. 3, p. 1, doi. 10.1177/26330040221139872

By:

Jain, Paridhi;
Badgujar, Lohit;
Spoorendonk, Jelle;
Buesch, Katharina

Publication type:

Article

Primary ovarian malignant mixed Müllerian tumor: a rare case report.

Published in:

Therapeutic Advances in Rare Disease, 2022, v. 3, p. 1, doi. 10.1177/26330040221107389

By:

Jagtap, Sunil V.;
Jagtap, Shubham S.;
Gudur, Rashmi;
Billawaria, Sonam

Publication type:

Article

Impact of the Covid-19 epidemic on a US sample of patients with myasthenia gravis.

Published in:

Therapeutic Advances in Rare Disease, 2022, v. 3, p. 1, doi. 10.1177/26330040221082673

By:

Gutierrez, Gloria;
Girma, Helen;
Kuhnell, Pierce;
Macaluso, Maurizio;
Kaminski, Henry J.

Publication type:

Article

Congenital disorder of glycosylation -- one size does not fit all: a parent's perspective.

Published in:

Therapeutic Advances in Rare Disease, 2022, v. 3, p. 1, doi. 10.1177/26330040221118099

By:

Feinberg, Konstantin

Publication type:

Article

Pharmacology update: pamidronate for hypertrophic pulmonary osteoarthropathy in palliative care.

Published in:

Therapeutic Advances in Rare Disease, 2022, v. 3, p. 1, doi. 10.1177/26330040211070298

By:

Faust, Bethany;
Parkinson, Aaron;
Baumrucker, Steven J.

Publication type:

Article

'Granulomatosis with polyangiitis after Pfizer vaccination': a case report.

Published in:

Therapeutic Advances in Rare Disease, 2022, v. 3, p. 1, doi. 10.1177/26330040221130084

By:

Essien, Francis;
Evans, Jordan;
Kyle, Andrew;
Urisman, Anatoly;
Adams, Nicholas

Publication type:

Article

Mannose treatment improves immune deficiency in mannose phosphate isomerase--congenital disorder of glycosylation: case report and review of literature.

Published in:

Therapeutic Advances in Rare Disease, 2022, v. 3, p. 1, doi. 10.1177/26330040211091283

By:

derik De Graef, Die;
Mousa, Jehan;
Waberski, Marta Biderman;
Morava, Eva

Publication type:

Article

Future of genetic therapies for rare genetic diseases: what to expect for the next 15 years?

Published in:

Therapeutic Advances in Rare Disease, 2022, v. 3, p. 1, doi. 10.1177/26330040221100840

By:

Braga, Luiza Amara Maciel;
Filho, Carlos Gilbert Conte;
Mota, Fabio Batista

Publication type:

Article

A Systematic Review investigating the Effectiveness of Exercise training in Glycogen Storage Diseases.

Published in:

Therapeutic Advances in Rare Disease, 2022, v. 3, p. 1, doi. 10.1177/26330040221076497

By:

Bordoli, Claire;
Murphy, Elaine;
Varley, Ian;
Sharpe, Graham;
Hennis, Philip

Publication type:

Article