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Live by the SORD: Applied Therapeutics looks to rare disease NDA

Based on positive phase III study data, Applied Therapeutics Inc. plans to take its CNS-penetrant aldose reductase inhibitor to the U.S. FDA to talk about an NDA for treating the rare disease sorbitol dehydrogenase (SORD) deficiency. SORD, a hereditary axonal neuropathy created by sorbitol dehydrogenase gene mutations, affects about 3,300 people in the U.S. and about 4,000 in Europe, according to Applied Therapeutics. ....

Therapeutics Inc , Applied Therapeutics , Applied Therapeutics Inc , At 007 , Sorbitol Dehydrogenase Deficiency , Rare Disease , Neurology Psychiatric , Charcot Marie Tooth Disease , Distal Hereditary Motor Neuropathy ,

Global eClinical Solutions Market Outlook 2024-2028: Industry to Reach $13.48 Billion by 2028, Driven by Interoperability and Telehealth Adoption

Dublin, Feb. 14, 2024 (GLOBE NEWSWIRE) The "eClinical Solutions Global Market Report 2024" report has been added to ResearchAndMarkets.com's offering.The global eClinical solutions market size has grown rapidly in recent years. It will grow from $7.59 billion in 2023 to $8.47 billion in 2024 at a compound annual growth rate (CAGR) of 11.6%. The growth observed in the historical period can be attributed to several key factors, including the expansion of healthcare digitalization, the widesprea ....

South Korea , United Kingdom , United States , Development Phase , Bioclinicia Inc , Maxisit Inc , Anju Software Inc , Medidata Solutions Inc , Studykik Inc , Acceliant Inc , Omnicomm Systems Inc , Clinical Research , Ai Inc , Medlink Inc , Cinven Ltd , Datatrak International Inc , Key Companies , Clinical Solutions , System Inc , Whealthcare Solutions Inc , Watson Health , Parexel International Corporation , Mednet Solutions , Saama Technologies Inc , Academic Research Institutions , Biopharmaceutical Companies ,

Kalvista's oral hereditary angioedema drug hits phase III goals

Kalvista Pharmaceuticals Inc. is promising to “change the paradigm” in the treatment of hereditary angioedema, after announcing positive phase III data for its oral on-demand kallikrein inhibitor sebetralstat. Rather than waiting four to five hours to self-administer therapy, as is the case for approved injectable on-demand therapies, there was a median time to dosing of 10 minutes after the start of an attack in the 136-patient placebo-controlled trial. ....

Kalvista Pharmaceuticals Inc , Kalvista Pharmaceuticals , Kalvista Pharmaceuticals Inc , Oral Kallikrein Inhibitor , Hereditary Angioedema , On Demand , Hae Attacks ,