Experimental Beta Thalassemia Drug Gains Temporary Short-Term Approval in Europe
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Experimental Beta Thalassemia Drug Gains Temporary Short-Term Approval in Europe

According to a publication from BNN Bloomberg, regulatory authorities in the European Commission recently granted conditional marketing authorization (CMA) to Zynteglo. Zynteglo is a gene therapy designed to treat beta…

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Very First Patient Dosed with Investigational Therapy for Friedreich’s Ataxia in Phase 2 Clinical Trial

Friedreich's Ataxia Friedreich's ataxia (FRDA) is a rare disease which is caused by a frataxin deficiency. This deficiency results in the degeneration of nerves within the spinal cord. This nerve…

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Let the Men in Your Life Know About Men’s Health and Rare Diseases for Father’s Day

June 10th through 16th is Men Health's/Rare Dads Week! We would like to devote some attention to the rare diseases that affect mostly men - because to quote Congressman Bill…

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The FDA, Congress, A Young Woman Dying of ALS, Her Physician, and Her Parents Are All Struggling Over Access to an Untested Therapy

  The Hermstads of Spencer, Iowa lost Jaci’s twin to ALS and now they must watch Jaci, rapidly descend into the depths of the same disease. Jaci has a rare…

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