New Collaboration Will Test Experimental Clinical Trial Designs for Stargardt Disease Drugs
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New Collaboration Will Test Experimental Clinical Trial Designs for Stargardt Disease Drugs

According to a story from pharmavoice.com, a new partnership between the biotechnology company Vitrisa Therapeutics and the Foundation Fighting Blindness Clinical Research Institute (FFB-CRI) is in the works in order…

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A New Study Aims to Improve Diagnoses for People with WHIM Syndrome
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A New Study Aims to Improve Diagnoses for People with WHIM Syndrome

A large clinical study will be carried out to develop a protocol for identifying a rare primary immunodeficiency disease called WHIM syndrome in patients, reports BioPortfolio. The Jeffrey Modell Foundation,…

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The UK Parliament Met With a Mother Advocating For Duchenne Muscular Dystrophy Patients
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The UK Parliament Met With a Mother Advocating For Duchenne Muscular Dystrophy Patients

Phillippa Farrant, whose son, Daniel, has been diagnosed with Duchenne muscular dystrophy (DMD), recently appeared in Parliament in the UK to fight for improved access to care and treatment for…

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A Gene Therapy Being Developed for Sanfilippo Syndrome Has Been Granted RMAT status in the US.
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A Gene Therapy Being Developed for Sanfilippo Syndrome Has Been Granted RMAT status in the US.

A gene therapy being developed as a treatment for Sanfilippo syndrome has just been awarded Regenerative Medicine Advanced Therapy (RMAT) status in the US, reports GlobalGenes. Sanfilippo syndrome is a…

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