Nikkie Miranda

130 actions

Action Alert. Tell the FDA to include Neurodegenerative Lysosomal Storage Diseases to the list of diseases included in the patient-focused drug initiative.

JJB, along with our sister organizations representing lysosomal storage diseases that affect the brain, such as Sanfilippo, are asking for your support. As many of you may recall, JJB was instrumental in adding specific language to PDUFA V. This was due to our grassroots call to action. The

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