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Children denied access to life-changing drug due to paperwork, say families

Summary by The Independent

Givinostat should be available to people with Duchenne muscular dystrophy through a type of compassionate use scheme.

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The Independent

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Children denied access to life-changing drug due to paperwork, say families

Givinostat should be available to people with Duchenne muscular dystrophy through a type of compassionate use scheme.

1 month ago·London, United Kingdom

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radiojackie.com

Families of children with a genetic muscle-wasting condition claim NHS failings are stopping them accessing a drug that slows it down.

Families of children with a genetic muscle-wasting condition claim NHS failings are stopping them accessing a drug that slows it down. The drug Givinostat was made available to patients with Duchenne Muscular Dystrophy through the Early Access Programme last year. But campaigners say clinical teams lack “the capacity to process the paperwork” to administer it. Una Farell is from the Hammersmith based charity, Duchenne UK.

1 month ago

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Muscular Dystrophy News

What superheroes look like in life with Duchenne muscular dystrophy

Superheroes were a large part of my life less than a decade ago. I am a boy mom. We had four sons in five years, and our house quickly became a lived-in museum of all things boys. Power Rangers, X-Men, and Marvel and DC Comics characters were like family members for a few years. Those superheroes joined our family of nine. I share seven children with my husband, Jason: Lexi, 23, Max, 19, Chance, 17, Rowen, 16, Charlie, 14, Mary, 10, and Callie, …

1 month ago

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ajmc.com

Survey Explores Needs of Patients With DMD, BMD Transitioning to Adulthood

Activities of daily life, education, and employment were areas of difficulty in the transition to adulthood for patients with Duchenne muscular dystrophy (DMD) or Becker muscular dystrophy (BMD), but those who had more siblings reported being more ready to transition to adult life.

1 month ago

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Rother Radio - Rotherham & Sheffield's Best Variety of Hits!

Children denied access to life-changing drug due to paperwork, say families - Rother Radio

The families of children with a severe genetic muscle-wasting condition claim NHS failings are preventing them from accessing a drug that slows its progression. Clinical teams lack the “capacity to process the paperwork” associated with administering givinostat, campaigners said. The drug was made available to eligible patients through a type of compassionate use scheme known as an Early Access Programme (EAP) last year. Parents described the si…

1 month ago

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Muscular Dystrophy Association

AFFINITY DUCHENNE: RGX-202 in Participants with Duchenne Muscular Dystrophy (DMD)

Register Activity Overview: RGX-202 is an investigational, one-time AAV gene therapy designed to deliver an optimized microdystrophin gene encoding for a novel microdystrophin protein that includes the C-Terminal (CT) domain found in naturally occurring dystrophin. AFFINITY DUCHENNE®, a phase I/II/III multi-center, open-label trial of a one-time intravenous administration of RGX-202 is enrolling ambulatory boys aged one and above with Duchenne t…

1 month ago

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