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argenx Receives Positive CHMP Opinion for Efgartigimod for the Treatment of Adult Patients with Generalized Myasthenia Gravis in Europe

Positive opinion based on Phase 3 ADAPT trial showing efgartigimod provided clinically meaningful improvements in strength and quality of life measuresEuropean Commission decision on marketing. | June 24, 2022 ....

United States , France General , Kelsey Kirk , Michelle Greenblatt , Beth Delgiacco , European Medicines Agency , European Commission , European Union Member States , Immunology Innovation Program , Committee For Medicinal Products Human Use , Medicinal Products , Human Use , Union Member States , Institut De Myologie , Myasthenia Gravis Activities , Daily Living , About Phase , North America , Argenx Se Stock Exchange , Press Release , N Argx Nl0010832176 ,

'It's a real-life Hunger Games': a lifesaving drug costs $2m, but not every child can get it | Pharmaceuticals industry

Zolgensma is a revolutionary gene therapy that can stop a deadly childhood condition called SMA in its tracks. It’s also one of the most expensive drugs in the world ....

City Of , United Kingdom , United States , France General , New South Wales , Mike Fraser , Elizabeth Wraige , Francesco Muntoni , Arnaud Robert , Elizabeth Warren , Martine Barkats , Bernie Sanders , Evelina London , Institut De Myologie , Paolo Woods , Happy Pills , Novartis Gene , Great Ormond Street ,

Treatabolome project designed to shorten diagnosis-to-treatment time for patients with rare diseases

The Treatabolome project is a research initiative to develop a freely available, interoperable online platform to disseminate rare disease and gene-specific treatment information to healthcare professionals regardless of their level of specialized expertise to reduce treatment delays. This initiative is highly relevant to neuromuscular disorders as they are rare diseases by definition. In this special issue of the Journal of Neuromuscular Diseases, experts contribute Treatabolome-feeding systematic literature reviews on rare neurological and neuromuscular disorders. ....

Noord Holland , France General , European Research , European Reference Networks , Centre De Recherche En Myologie , Research Project , Neuromuscular Diseases , Guest Editor Gis , Sorbonne Universit , Institut De Myologie , Centre De Recherche , Rare Neurological Diseases , Rare Neuromuscular Diseases , Genome Phenome Analysis Platform , Reference Networks , Medicine Health , நூற்த் ஹாலண்ட் , பிரான்ஸ் ஜநரல் , ஐரோப்பிய ஆராய்ச்சி , ஐரோப்பிய குறிப்பு நெட்வொர்க்குகள் , ஆராய்ச்சி ப்ராஜெக்ட் , நரம்புத்தசை நோய்கள் , மையம் டி ரெக்ஃப்‌ச் , ரேர் நரம்பியல் நோய்கள் , குறிப்பு நெட்வொர்க்குகள் , மருந்து ஆரோக்கியம் ,

New initiative to reduce treatment delays for patients with rare diseases

The Treatabolome project is a research initiative to develop a freely available, interoperable online platform dedicated to disseminating rare disease and gene-specific treatment information to healthcare professionals regardless of their level of specialized expertise. ....

France General , Emily Henderson , European Research , European Reference Networks , Centre De Recherche En Myologie , Research Project , Neuromuscular Diseases , Guest Editor , Sorbonne Universit , Institut De Myologie , Centre De Recherche , Rare Neurological Diseases , Rare Neuromuscular Diseases , Genome Phenome Analysis Platform , Reference Networks , Rare Disease , Research Project , பிரான்ஸ் ஜநரல் , எமிலி ஹென்டர்சன் , ஐரோப்பிய ஆராய்ச்சி , ஐரோப்பிய குறிப்பு நெட்வொர்க்குகள் , ஆராய்ச்சி ப்ராஜெக்ட் , நரம்புத்தசை நோய்கள் , விருந்தினர் ஆசிரியர் , மையம் டி ரெக்ஃப்‌ச் , ரேர் நரம்பியல் நோய்கள் ,