Heartbreaking NHS funding blow to sick children like Ollie and Amelia

EXCLUSIVE: NICE decision means kids diagnosed with devastating neurodegenerative condition will no longer receive life-lengthening enzyme therapy on NHS ...
Baltimore Fishbowl

Batten disease conference

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Tyneside mum hits out at 'unthinkable' decision not to approve 'transformational' drug for rare genetic disease

The Rich family have seen first hand how much the drug can help children with a devastating Batten disease diagnosis ...

Polaryx Therapeutics Marks Rare Disease Day, Reaffirming Commitment to Patients with Rare Pediatric Lysosomal Storage Disorders

Polaryx Therapeutics (Nasdaq: PLYX), a clinical-stage biotechnology company developing novel, disease-modifying therapies for rare, pediatric lysosomal storage disorders (“LSDs”), joins the global ...
Des Moines Register

Sweet 16 Celebration Becomes Lifeline for Twins Battling Rare Disease

This campaign turns a birthday celebration into a chance to help children around the world. Every bid and every donation helps move research forward.” — Carol Schwimmer, ForeBatten Foundation CHAPEL ...
Wicked Local

Collaborations Pharmaceuticals, Inc. demonstrates a treatment for CLN1 Batten disease crosses the blood-brain-barrier

rhPPT1 is a recombinant protein used to treat an ultra-rare disease called Batten disease CLN1 for which there is currently no FDA approved treatment available. These findings suggest intravenous ...