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Novartis to proceed with phase 3 trial for intrathecal OAV-101 in older SMA patients

Novartis to proceed with phase 3 trial for intrathecal OAV-101 in older SMA patients
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Shephard Mpofu , Drug Administration , Hammersmith Functional Motor Scale Expanded , Novartis Gene Therapies , ஷெப்பர்ட் ம்போபு , சுத்தியல் செயல்பாட்டு மோட்டார் அளவு விரிவாக்கப்பட்டது , நோவர்த்திச் கீந் சிகிச்சைகள் ,

Results from the Second Year of Evrysdi™ (risdiplam) Treatment Demonstrated Sustained Improvement of Motor Function in a Broad Range of SMA Patients

Results from the Second Year of Evrysdi™ (risdiplam) Treatment Demonstrated Sustained Improvement of Motor Function in a Broad Range of SMA Patients
- Sustained or improved motor function was observed with 24 months of treatment -
- Patients and caregivers reported a greater ability to complete activities of daily livingwith increased independence -
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SOUTH PLAINFIELD, N.J., March 16, 2021 /PRNewswire/ PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced two-year data from Part 2 of the SUNFISH trial evaluating Evrysdi™ (risdiplam) in children and adults with Type 2 or Type 3 spinal muscular atrophy (SMA) at the 2021 Muscular Dystrophy Association (MDA) Virtual and Scientific Conference. These results demonstrated that Evrysdi patients sustained or improved in motor function after 24 months of treatment. Furthermore, the patients and caregivers reported improvements in their ability to function i ....

United States , Kylie Okeefe , Stuartw Peltz , Jane Baj , Muscular Dystrophy Association , Toddler Development , Drug Administration , Therapeutics Inc , Roche Group , Dystrophy Association , Chief Executive Officer , Motor Function Measure , Revised Upper Limb Module , Hammersmith Functional Motor Scale Expanded , Evrysdi Clinical , Gross Motor Scale , Bayley Scales , Spinal Muscular Atrophy , More Information , Looking Statements , Private Securities Litigation Reform Act , Annual Report , Ptc Therapeutics , ஒன்றுபட்டது மாநிலங்களில் , கைலி ஓக்கீபே , ஜேன் பஜ் ,

F. Hoffmann-La Roche Ltd: New two-year data show Roche's Evrysdi (risdiplam) continues to demonstrate improvement or maintenance of motor function in people aged 2-25 with Type 2 or Type 3 Spinal Muscular Atrophy (SMA)

F. Hoffmann-La Roche Ltd: New two-year data show Roche's Evrysdi (risdiplam) continues to demonstrate improvement or maintenance of motor function in people aged 2-25 with Type 2 or Type 3 Spinal Muscular Atrophy (SMA)
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United States , Nathalie Meetz , Levi Garraway , Daniel Grotzky , Jon Kaspar Bayard , Karl Mahler , Sabine Borngr , Gerard Tobin , Bruno Eschli , Birgit Masjost , Lisa Tuomi , Eugenio Mercuri , Loren Kalm , Patrick Barth , Karsten Kleine , Muscular Dystrophy Association , Toddler Development , Department Of Pediatric Neurology , Drug Administration , Head Of Global Product Development , European Medicines Agency , Roche Group , Pharmaceuticals Industry , Committee For Medicinal Products Human Use , Catholic University , Roche Group Media Relations ,

New two-year data show Roche's Evrysdi (risdiplam) continues to demonstrate improvement or maintenance of motor function in people aged 2-25 with Type 2 or Type 3 Spinal Muscular Atrophy (SMA) Swiss Stock Exchange:RO

New two-year data show Roche's Evrysdi (risdiplam) continues to demonstrate improvement or maintenance of motor function in people aged 2-25 with Type 2 or Type 3 Spinal Muscular Atrophy (SMA) Swiss Stock Exchange:RO
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United States , Nathalie Meetz , Levi Garraway , Daniel Grotzky , Eugenio Mercuri , Patrick Barth , Karsten Kleine , Muscular Dystrophy Association , Toddler Development , Department Of Pediatric Neurology , Drug Administration , Head Of Global Product Development , European Medicines Agency , Roche Group , Pharmaceuticals Industry , Catholic University , Roche Group Media Relations , Committee For Medicinal Products Human Use , Dystrophy Association , Virtual Clinical , Scientific Conference , Pediatric Neurology , Motor Function Measure , Revised Upper Limb Module , Hammersmith Functional Motor Scale Expanded , Chief Medical Officer ,