Results 81 to 90 of about 1,115 (145)

Neurovisceral Syndrome in a Patient with Monoclonal Gammopathy of Undetermined Significance: A Confirmed Case of Variegate Porphyria. [PDF]

Cureus
Fatima I, Awais KAD, Sehbai A, Tareen AAK, Shaharyar M.   +4 more
europepmc   +1 more source

Platform Assessment of Concentration-QTc Relationship Across GalNAc-siRNA Molecules. [PDF]

Clin Pharmacokinet
Badri P, Kolachana K, Duong A, Lasko M, Nandi T, Mehrotra N, Robbie GJ.   +6 more
europepmc   +1 more source

A PHASE 1/2 OPEN LABEL EXTENSION STUDY OF GIVOSIRAN, AN INVESTIGATIONAL RNAI THERAPEUTIC, IN PATIENTS WITH ACUTE INTERMITTENT PORPHYRIA

Hematology, Transfusion and Cell Therapy, 2020
R. Marchi, L. Duarte, S. P, R. D, A. Ke, B. Hl, S. E, H. P, B. M, E. Al   +9 more
doaj   +1 more source

C16-siRNAs in Focus: Development of ALN-APP, a Promising RNAi-Based Therapeutic for Alzheimer's Disease. [PDF]

Pharmaceuticals (Basel)
Titze-de-Almeida R, Oliveira Gomes GM, Santos TCD, Titze-de-Almeida SS.   +3 more
europepmc   +1 more source

Porphyrias: Pathophysiology and clinical management recommendations for hepatologists. [PDF]

Hepatol Commun
Ricci A, Corradini E, Buzzetti E, Pietrangelo A, Pietrangelo A, Ventura P, Modena Centre of Rare Diseases - Porphyria Working Group.   +6 more
europepmc   +1 more source

siRNA Therapeutics for the Treatment of Hereditary Diseases and Other Conditions: A Review. [PDF]

Int J Mol Sci
Shevelev A, Pozdniakova N, Generalov E, Tarasova O.   +3 more
europepmc   +1 more source

Acute hepatic porphyrias. [PDF]

Porto Biomed J
Marques I, Marcos P.
europepmc   +1 more source

TWELVE-MONTH INTERIM ANALYSIS OF EFFICACY AND SAFETY OF GIVOSIRAN, AN INVESTIGATIONAL RNAI THERAPEUTIC FOR ACUTE HEPATIC PORPHYRIA, IN THE ENVISION OPEN LABEL EXTENSION

Hematology, Transfusion and Cell Therapy, 2020
R. Marchi, L. Duarte, S. E, B. M, R. Dc, S. P, S. U, A.P. P, B. Dm, E. Al, K. S, P. C, P. Jd   +12 more
doaj   +1 more source

Givosiran for the treatment of acute hepatic porphyria

Future Rare Diseases
Julian Pohl, Frank Tacke
openaire   +1 more source

Hyponatremia and Abdominal Pain: A Case Report of Acute Hepatic Porphyria. [PDF]

Cureus
Roldão M, Soares Dionísio A, Duarte J, Salgadinho Machado A, Anastácio M.   +4 more
europepmc   +1 more source