Positive Trial Data is Welcomed News for Spinal Muscular Atrophy Patients

Spinal muscular atrophy (SMA) is a rare disease causing progressive muscular weakness. Severe forms of the disease can be fatal to infants. Apitegromab is an experimental therapy that was successful…

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Investigational Therapy Earns Orphan Drug Designation for Hereditary Angioedema
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Investigational Therapy Earns Orphan Drug Designation for Hereditary Angioedema

According to a story from Business Wire, the biopharmaceutical company Astria Therapeutics, Inc., recently announced that its experimental therapy navenibart has earned the US Food and Drug Administration's Orphan Drug…

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NDA for NF1-PN Treatment Charges Ahead with Priority Review
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NDA for NF1-PN Treatment Charges Ahead with Priority Review

According to a press release from GlobeNewswire, the biopharmaceutical company SpringWorks Therapeutics recently announced that the US Food and Drug Administration (FDA) has granted the company's New Drug Application (NDA)…

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These Nonprofits Just Shelled Out $5.2 Million to Fund An Experimental Parkinson’s Drug
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These Nonprofits Just Shelled Out $5.2 Million to Fund An Experimental Parkinson’s Drug

According to a story from PR Newswire, the biotech company Mission Therapeutics was just awarded $5.2 million in funding to support MTX325, the company's investigational Parkinson's disease therapy. Intended to…

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Japanese Scientists Reversed Signs of Alzheimer’s by Restoring Synapse Function in Mouse Model
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Japanese Scientists Reversed Signs of Alzheimer’s by Restoring Synapse Function in Mouse Model

A team of scientists from Japan appear to have pioneered a method for reversing synapse damage in an Alzheimer's disease mouse model. Although encouraging, the theory must be tested in…

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FDA’s Panel of Experts Vote 11-0 in Favor of Donanemab to Treat Alzheimer’s Disease
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FDA’s Panel of Experts Vote 11-0 in Favor of Donanemab to Treat Alzheimer’s Disease

The idiom ‘third time’s a charm’ may have some significance in this instance. The panel that advises the FDA just voted unanimously in favor of Eli Lilly’s experimental drug, donanemab.…

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