La NHS conclut un accord pour l’accès à un médicament essentiel contre l’atrophie musculaire spinale … pour le moment
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La NHS conclut un accord pour l’accès à un médicament essentiel contre l’atrophie musculaire spinale … pour le moment

Selon un article de BBC News, le père Shakil Malji demande depuis des mois que sa jeune fille Maryam, atteinte d’une maladie rare et mortelle appelé l’atrophie musculaire spinale, puisse…

Continue Reading La NHS conclut un accord pour l’accès à un médicament essentiel contre l’atrophie musculaire spinale … pour le moment
England’s NHS Reaches Agreement for Coverage with SMA Drug Manufacturer Biogen
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England’s NHS Reaches Agreement for Coverage with SMA Drug Manufacturer Biogen

According to a publication from Express Digest, Britain's National Health Service (NHS) has finally reached an agreement with American biotechnology company Biogen Inc. over the price of Spinraza - a…

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Experimental Treatment for Multiple Myeloma Earns Orphan Drug Designation
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Experimental Treatment for Multiple Myeloma Earns Orphan Drug Designation

According to a story from globenewswire.com, the biopharmaceutical company Poseida Therapeutics, Inc. recently announced that the company's investigational product candidate P-BCMA-101 has been granted Orphan Drug Designation from the US…

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This Spinocerebellar Ataxia Patient is Committed to Spreading Awareness
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This Spinocerebellar Ataxia Patient is Committed to Spreading Awareness

According to a story from Kamloops Coffee Talk, Darrin Rein, of Kamloops, British Columbia, was just recently diagnosed with spinocerebellar ataxia, a rare disease that affects mobility and coordination. Despite…

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Could Stopping Using the Word “Rare” Increase the Rate of Diagnosis for Rare Disease Patients?

The Word "Rare" Unfortunately, many experts in rare disease believe the phrase "rare disease" may be harming the very population it describes. The Director of the National Center for Advancing…

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