Last Posted: Sep 22, 2020
- Graphical Analyses in the Regulatory Evaluation of Gene Therapy Applications.
Lin Xue et al. Therapeutic innovation & regulatory science 2020 Sep
- Current Perspectives and Unmet Needs of Primary Immunodeficiency Care in Asia Pacific.
Leung Daniel et al. Frontiers in immunology 2020 111605
- Use of the Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) in X-Linked Myotubular Myopathy: Content Validity and Psychometric Performance.
Duong Tina et al. Journal of neuromuscular diseases 2020 Sep
- An assessment of the continuing medical education needs of US physicians in the management of patients with beta thalassemia.
Stacy Sylvie et al. Annals of hematology 2020 Sep
- Genetic testing for inherited eye conditions in over 6,000 individuals through the eyeGENE network.
Goetz Kerry E et al. American journal of medical genetics. Part C, Seminars in medical genetics 2020 Sep
- Overview of Gene Therapy in Spinal Muscular Atrophy and Duchenne Muscular Dystrophy.
Abreu Nicolas J et al. Pediatric pulmonology 2020 Sep
- Ophthalmic genetics practice and research in India: Vision in 2020.
Bansal Mayank et al. American journal of medical genetics. Part C, Seminars in medical genetics 2020 Aug
- Primary Immunodeficiencies in Russia: Data From the National Registry.
Mukhina Anna A et al. Frontiers in immunology 2020 111491
- Curative gene therapies for rare diseases.
Maldonado Rocio et al. Journal of community genetics 2020 Aug
- Toward the Clinical Application of Therapeutic Angiogenesis Against Pediatric Ischemic Retinopathy.
Jo Dong Hyun et al. Journal of lipid and atherosclerosis 2020 May 9(2) 268-282
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