The National Organization for Rare Disorders (NORD) says it’s “disappointed and dismayed” after the House of Representatives voted 227-205 last week to repeal the Orphan Drug Tax Credit as part of a U.S. tax reform package. A similar package before the Senate Finance Committee does not repeal the credit…
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Batten disease may be caused by the loss of synaptic connections between neurons in the brain, according to a study that detailed abnormalities in the brains of mice, lacking the Cln3 gene. These mice also lacked a certain type of neuron crucial for building neural networks. Researchers at Jena University…
WASHINGTON — As the Batten Disease Support and Research Association celebrates its 30th anniversary, the group’s executive director says scientists are edging ever closer to treatments for the incurable genetic disorder. Margie Frazier said the organization she’s headed for five years is the only one that represents all 14 forms…
The Beyond Batten Disease Foundation has started a Be A Hero campaign to raise $1 million and public awareness about the form of the disease that strikes youngsters. Craig and Charlotte Benson founded the organization in 2008 after doctors diagnosed their then-6-year-old daughter Christiane with juvenile Batten disease. Now 15,…
Children’s National Health System no longer treats just kids. Its Rare Disease Institute, launched in April 2017, has partnered with the National Organization for Rare Disorders (NORD) to become the first of many U.S. “centers of excellence” to look after patients with rare diseases, regardless of age. The effort…
Glial cell function is impaired in the juvenile form of Batten disease, leading to nerve cell loss, a mouse study has found. The study, “Glial cells are functionally impaired in juvenile neuronal ceroid lipofuscinosis and detrimental to neurons,” was appeared in the journal Acta Neuropathologica Communications. Glial cells,…
More therapies are now available for the 30 million or so people with rare diseases in the U.S. than ever before, and millions of dollars are being invested in clinical studies that will test new ways of evaluating — and advancing — potential treatments, including the use of natural history…
Brineura (cerliponase alfa), the first therapy approved to treat children with Batten disease, received the 2017 “Best of What’s New” award in the health category by Popular Science, a science and technology magazine. Brineura is BioMarin’s therapeutic product developed to treat the underlying cause of tripeptidyl…
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#NORDsummit – Despite Criticism, Orphan Drug Act Is Working to Advance Needed Treatments, FDA Says
As Congress begins debate this week to overhaul the U.S. tax code, lawmakers should leave the Orphan Drug Act (ODA) — and the tax incentives it offers pharmaceutical companies to develop therapies for rare diseases — off the table. That’s the message being pushed by the National Organization for…
Gene therapy is able to reduce toxic protein aggregates in a zebrafish model of one form of adult-onset Batten disease, a new study from China shows. The study, “Gene Therapy of Adult Neuronal Ceroid Lipofuscinoses with CRISPR/Cas9 in Zebrafish,” appeared in the journal Human Gene Therapy. The study’s…
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