ATYR1923 Safe and Effective for Pulmonary Sarcoidosis
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ATYR1923 Safe and Effective for Pulmonary Sarcoidosis

Clinical trials can be helpful tools in determining the safety, efficacy, tolerability, recommended dose, and pharmacokinetics of specific drugs (among other things). In doing so, these trials help tell researchers…

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The Secret Sauce for Bringing Innovative Therapies to Patients with Rare Diseases Faster
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The Secret Sauce for Bringing Innovative Therapies to Patients with Rare Diseases Faster

Written by Lori Lawter, MPH We’ve heard a lot about bringing drugs to market lately, especially in light of the COVID-19 vaccine.  You might wonder, “Is there a ‘secret sauce’…

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PTC-AADC Shows Benefits for Patients with AADC Deficiency, Data Shows

Recently, biopharmaceutical company PTC Therapeutics, Inc. performed a five-year data analysis to evaluate the continued safety, efficacy, and tolerability of PTC-AADC. The company developed this novel gene therapy to improve…

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Barth Syndrome Chairman Appeals to the FDA For Appropriate Guidelines When Approving Drugs for Ultra-Rare Diseases

According to an article submitted to StatNews by members of the Barth Syndrome Foundation, the FDA approved the drug Aduhelm in accordance with its accelerated approval program. Although the treatment…

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Phase 2 Trial Results Show Loncastuximab Tesirine as Beneficial for DLBCL
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Phase 2 Trial Results Show Loncastuximab Tesirine as Beneficial for DLBCL

In the Phase 2 LOTIS-2 clinical trial, researchers evaluated the safety, efficacy, and tolerability of Loncastuximab tesirine for patients with aggressive diffuse large B-cell lymphoma (DLBCL). While there are treatment…

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