பெக்கர் தசை டிஸ்ட்ரோபி News Today : Breaking News, Live Updates & Top Stories | Vimarsana

Stay updated with breaking news from பெக்கர் தசை டிஸ்ட்ரோபி. Get real-time updates on events, politics, business, and more. Visit us for reliable news and exclusive interviews.

Top News In பெக்கர் தசை டிஸ்ட்ரோபி Today - Breaking & Trending Today

Italfarmaco Announces Topline Data from Phase 2 Trial with Givinostat in Patients with Becker Muscular Dystrophy


Posted on
8181
Italfarmaco Group announced today topline data from its proof-of-concept Phase 2 trial with Givinostat, the company’s proprietary histone deacetylase (HDAC) inhibitor, in 51 adult males with Becker Muscular Dystrophy (BMD). The study was designed to evaluate the effect of Givinostat in BMD, building on the experience developed in Duchenne Muscular Dystrophy. Based on this experience, change in total fibrosis in the muscle biopsy was selected as the primary endpoint of the study and change in fat fraction and contractile CSA using quantitative Magnetic Resonance Imaging (MRI) as key secondary endpoints. Givinostat did not show significant difference in the primary endpoint compared to placebo. However, significant difference from placebo in MRI of muscles in the whole thigh as well as quadriceps confirmed the ability of Givinostat to counteract muscle deterioration in the treated BMD patients. Th ....

San Diego , United States , Regione Lombardia , Paolo Bettica , Krista Vandenborne , European Regional Development Fund , Santa Lucia Foundation , University Of Florida , Department Of Physical Therapy , Regional Operational Program , Italfarmaco Group , Neurologist At Leiden University Medical Centre , University Of Milan , Becker Muscular Dystrophy , Duchenne Muscular , Magnetic Resonance Imaging , Parent Project Muscular Dystrophy , Muscular Dystrophy , Chief Medical Officer , Duchenne Muscular Dystrophy , Transfer Domain , Motor Function Measure , Adverse Events , Treatment Emergent Adverse Events , Principal Investigator , Physical Therapy ,

(SRRK) - Scholar Rock Reports Apitegromab Data In Spinal Muscular Atrophy

(SRRK) - Scholar Rock Reports Apitegromab Data In Spinal Muscular Atrophy
benzinga.com - get the latest breaking news, showbiz & celebrity photos, sport news & rumours, viral videos and top stories from benzinga.com Daily Mail and Mail on Sunday newspapers.

Scholar Rock , Revised Hammersmith Scale , Becker Muscular Dystrophy , அறிஞர் பாறை , பெக்கர் தசை டிஸ்ட்ரோபி ,

PTC Presents Results from a Real-World Study of Steroid Switching in the Treatment of Patients with Dystrophinopathies

PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced results from a real-world study of patients with Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD) who switched from prednisone to EMFLAZA® (deflazacort) following the FDA approval. The primary reason the majority of patients chose to switch to EMFLAZA was a desire to delay disease progression to improve the benefit and tolerability. During the 6-months average follow-up after switching, physician-reported outcomes were consistent with EMFLAZA addressing the primary reasons for switching. Data were presented at the 2021 Muscular Dystrophy Association (MDA) Virtual Clinical and Scientific Conference. ....

Duchenne Muscular Dystrophy , Ptc Therapeutics , Muscular Dystrophy Association , Slow Disease Progression , Becker Muscular Dystrophy , Muscular Dystrophy , ட சிகிச்சை , தசை டிஸ்ட்ரோபி சங்கம் , மெதுவாக நோய் ப்ரோக்ரெஶந் , பெக்கர் தசை டிஸ்ட்ரோபி , தசை டிஸ்ட்ரோபி ,