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1.
Assessment of the Impact of RNase in Patients With Severe Fatigue Related to Post-Acute Sequelae of SARS-CoV-2 Infection (PASC): A Randomized Phase 2 Trial of RSLV-132.
Clin Infect Dis;
2024 May 10.
Artículo
en Inglés
| MEDLINE | ID: mdl-38728385
2.
Long-term multisystemic efficacy of migalastat on Fabry-associated clinical events, including renal, cardiac and cerebrovascular outcomes.
J Med Genet;
60(7): 722-731, 2023 07.
Artículo
en Inglés
| MEDLINE | ID: mdl-36543533
3.
Assessment of plasma lyso-Gb3 for clinical monitoring of treatment response in migalastat-treated patients with Fabry disease.
Genet Med;
23(1): 192-201, 2021 01.
Artículo
en Inglés
| MEDLINE | ID: mdl-32994552
4.
Correction: Assessment of plasma lyso-Gb3 for clinical monitoring of treatment response in migalastat-treated patients with Fabry disease.
Genet Med;
23(1): 238, 2021 Jan.
Artículo
en Inglés
| MEDLINE | ID: mdl-33219325
5.
Corrigendum to "Long-term follow-up of renal function in patients treated with migalastat for Fabry disease" [Bichet et al., MGM Reports; 28 (2021) 100786].
Mol Genet Metab Rep;
29: 100807, 2021 Dec.
Artículo
en Inglés
| MEDLINE | ID: mdl-34934630
6.
Long-term follow-up of renal function in patients treated with migalastat for Fabry disease.
Mol Genet Metab Rep;
28: 100786, 2021 Sep.
Artículo
en Inglés
| MEDLINE | ID: mdl-34401344
7.
Wound closure in epidermolysis bullosa: data from the vehicle arm of the phase 3 ESSENCE Study.
Orphanet J Rare Dis;
15(1): 190, 2020 07 21.
Artículo
en Inglés
| MEDLINE | ID: mdl-32693833
8.
Use of the patient-reported outcomes measurement information system (PROMIS®) to assess late-onset Pompe disease severity.
J Patient Rep Outcomes;
4(1): 83, 2020 Oct 09.
Artículo
en Inglés
| MEDLINE | ID: mdl-33034771
9.
Efficacy and tolerability of the investigational topical cream SD-101 (6% allantoin) in patients with epidermolysis bullosa: a phase 3, randomized, double-blind, vehicle-controlled trial (ESSENCE study).
Orphanet J Rare Dis;
15(1): 158, 2020 06 23.
Artículo
en Inglés
| MEDLINE | ID: mdl-32576219
10.
Treatment of Autosomal Dominant Hypocalcemia Type 1 With the Calcilytic NPSP795 (SHP635).
J Bone Miner Res;
34(9): 1609-1618, 2019 09.
Artículo
en Inglés
| MEDLINE | ID: mdl-31063613
11.
Meta-analysis of the efficacy of a single dose of phenylephrine 10 mg compared with placebo in adults with acute nasal congestion due to the common cold.
Clin Ther;
29(6): 1057-70, 2007 Jun.
Artículo
en Inglés
| MEDLINE | ID: mdl-17692721
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