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Duchenne foundation funds first-in-human clinical study

According to RemedyDuchenne, a company devoted to furthering analysis and look after Duchenne muscular dystrophy (DMD), Kevin Flanigan (director of the Center for Gene Therapy) has used gene remedy to deal with two sufferers carrying the uncommon Duplication 2 mutation. The group reportedly started Flanigan’s early work within the space 10 years in the past, persevering with via the event’s preclinical part.

Outsourcing-Pharma (OSP) lately talked with RemedyDuchenne CEO and founder Debra Miller (DM) about the latest Duplication 2 study, the group’s mission to achieve a remedy for the uncommon and lethal illness, and her private expertise with DMD.a

OSP: Please inform us about RemedyDuchenne—who you’re, what you do, and the applications and initiatives you’ve received occurring.

DM: RemedyDuchenne is the biggest Duchenne group, together with applications in analysis, affected person care and assist, biobank providers and advocacy.  Our mission is our title; to remedy Duchenne muscular dystrophy.

RemedyDuchenne has offered funding to 15 initiatives which have progressed to human clinical trials, two of them receiving FDA approval.  We use a enterprise philanthropy mannequin with invests in biotech corporations and re-deploy proceeds from profitable investments, again into additional analysis.

OSP: Could you please inform us about Duchenne, and concerning the Duplication 2 mutation?

DM: Duchenne is brought on by a defect on the dystrophin gene, the biggest gene within the physique.  When a number of exons (items of DNA that should match collectively to make the dystrophin protein) are deleted, duplicated or in any other case out of order, the protein making mechanism stops.

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