Results 201 to 210 of about 12,392,515 (266)

Gene therapy for hemophilia B: results from the phase 1/2 101HEMB01/02 studies. [PDF]

Blood Adv
Pipe S, Poma A, Rajasekhar A, Everington T, Sankoh S, Allen J, Cataldo J, Crombez E.   +7 more
europepmc   +1 more source

Population Modeling of Factor IX Activity Following Administration of Fidanacogene Elaparvovec Gene Therapy in Participants with Hemophilia B. [PDF]

Clin Pharmacokinet
Wojciechowski J, Gaitonde P, Hughes JH, Ravva P.   +3 more
europepmc   +1 more source

Natural history of preexisting AAV5 antibodies in adults with hemophilia B during the lead-in of the etranacogene dezaparvovec phase 3 study. [PDF]

Mol Ther Methods Clin Dev
Klamroth R, Recht M, Key NS, Miesbach W, Pipe SW, Kaczmarek R, Drelich D, Salazar B, Le Quellec S, Monahan PE, Galante N, van der Valk P, Tarrant J.   +12 more
europepmc   +1 more source

A precision gene-engineered B cell medicine producing sustained levels of active factor IX for hemophilia B therapy. [PDF]

Mol Ther
Liu H, Singh S, Mullen TJ, Bullock C, Keegan S, Patterson T, Thakur S, Lundberg A, Shenker S, Couto R, Yadav C, Dastagir S, Li L, Bainter W, Liberzon E, Malloy CR, Lazzarotto CR, Ohsumi TK, Chilakala S, Chen HM, Kshirsagar R, Hohmann AF, Arlauckas SP, Lazorchak A, Scull C, Morgan RA.   +25 more
europepmc   +1 more source

Identification of Novel <i>F9</i> Gene Variants in 143 Vietnamese Patients with Hemophilia B. [PDF]

J Blood Med
Bach KQ, Duong CQ, Vu HTB, Nguyen BTN, Nguyen TT, Nguyen MT, Li R, Hutchison W, Esaq FS, Tran H, Nguyen TH.   +10 more
europepmc   +1 more source

Completion of phase 2b trial of etranacogene dezaparvovec gene therapy in patients with hemophilia B over 5 years. [PDF]

Blood Adv
von Drygalski A, Gomez E, Giermasz A, Castaman G, Key NS, Lattimore SU, Leebeek FWG, Miesbach WA, Recht M, Monahan PE, Le Quellec S, Pipe SW.   +11 more
europepmc   +1 more source

Total Hip Arthroplasty for Avascular Necrosis in a Patient With Hemophilia B. [PDF]

Arthroplast Today
Dandamudi S, Tseng J, Ratz J, Boggio L, Levine BR.   +4 more
europepmc   +1 more source

Erratum to "Relationship between Dose, Factor IX Activity Levels and Bleeding Probability for rIX-FP Prophylaxis in Hemophilia B: A Repeated Time-to-Event Analysis". [PDF]

Clin Pharmacol Ther
europepmc   +1 more source

Reproduction in Hemophilia

, 1983
Francis, Robert B, Kasper, Carol K.
core  

Segregation analysis of hemophilia A and B.

American journal of human genetics, 1985
We analyzed a sample of 1,485 families with hemophilia A and B and with unknown diagnosis. The frequency of sporadic cases was estimated to be .166 and .078 for the two types of hemophilia, respectively. The sex ratio of mutation rates did not differ significantly from unity. The average age of maternal grandfathers of probands at birth of mothers with
BARRAI, Italo Enrico, Cann H. M., Cavalli–Sforza L. L., BARBUJANI, Guido, De Nicola P.   +4 more
openaire   +3 more sources