Cure Rare Disease Expands Leadership Team to Accelerate Treatments for Ultra-Rare Conditions

Cure Rare Disease (CRD), a pioneering nonprofit biotechnology organization, has announced the addition of new scientific and strategic leaders, marking a significant step forward in its mission to develop therapies…

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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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