Autologous myoblast transplantation for oculopharyngeal muscular dystrophy: A phase I/IIa clinical study

Molecular Therapy

Volumn 22, Issue 1, 2014, Pages 219-225

  Périé, Sophie ^a,b,c,d   Trollet, Capucine ^b,c,d   Mouly, Vincent ^b,c,d   Vanneaux, Valérie ^e   Mamchaoui, Kamel ^b,c,d   Bouazza, Belaïd ^b,c,d   Marolleau, Jean Pierre ^f   Laforêt, Pascal ^g   Chapon, Françoise ^h,i   Eymard, Bruno ^g   Butler Browne, Gillian ^b,c,d   Larghero, Jérome ^e   St Guily, Jean Lacau ^a,b,c,d  

^a HÔPITAL TENON   (France)

^b SORBONNE UNIVERSITÉ   (France)

^c PITIÉ SALPÊTRIÈRE HOSPITAL   (France)

^d CNRS   (France)

^e SAINT LOUIS HOSPITAL   (France)

^f AMIENS UNIVERSITY HOSPITAL   (France)

^g ASSISTANCE PUBLIQUE HÔPITAUX DE PARIS   (France)

^h CAEN UNIVERSITY HOSPITAL   (France)

ⁱ INSERM U1075   (France)

Author keywords

[No Author keywords available]

Indexed keywords

ADULT; AGED; ARTICLE; AUTOLOGOUS MYOBLAST TRANSPLANTATION; CELL PROLIFERATION; CELL TRANSPLANTATION; CELL VIABILITY; CLINICAL ARTICLE; COMPARATIVE STUDY; DYSPHAGIA; FEMALE; FLUOROSCOPY; FOLLOW UP; GENETIC DISORDER; HUMAN; LIMB WEAKNESS; MALE; MCHORNEY SCORE; MYOTOMY; OCULOPHARYNGEAL MUSCULAR DYSTROPHY; PHARYNGEAL MUSCLE; PHASE 1 CLINICAL TRIAL; PHASE 2 CLINICAL TRIAL; PTOSIS; QUADRICEPS FEMORIS MUSCLE; QUALITY OF LIFE INDEX; RECURRENT LARYNGEAL NERVE PALSY; SAFETY; SALASSA GRADE; SCORING SYSTEM; STERNOCLEIDOMASTOID MUSCLE; SWALLOWING; TELOMERE HOMEOSTASIS; UPPER ESOPHAGUS SPHINCTER; VIDEOENDOSCOPY; VIDEOFLUOROSCOPY;

AGED; ESOPHAGEAL SPHINCTER, UPPER; FEMALE; HUMANS; MALE; MIDDLE AGED; MUSCULAR DYSTROPHY, OCULOPHARYNGEAL; MYOBLASTS, SKELETAL; PHARYNGEAL MUSCLES; TRANSPLANTATION, AUTOLOGOUS; TREATMENT OUTCOME;

EID: 84891829587     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2013.155     Document Type: Article

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