Taylor’s Tale Co-founder Races to ‘Change the World’ for Batten Disease Community
Laura King Edwards ran a half-marathon as a fifth anniversary nod to her sister’s first race — a 5K school run in 2008 that would ... Read more
Updates
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Extracellular Vesicles May Enhance Delivery of TPP1 to the Brain for Replacement Therapy in CLN2, Early Study Suggests
Extracellular vesicles derived from a type of immune cell called macrophage may hold potential as a less-invasive delivery system of TPP1 — the enzyme ... Read more -
At NY Genome Center, Legal Expert Presents Ethical Dilemmas in Gene Editing
Developing gene therapies for rare diseases is one thing. Creating gene-edited “designer babies” is quite another. German legal expert Timo Minssen outlined the potentially ... Read more -
Can Patients and Families Really Drive Therapy Development?
The therapy development process is a marathon, not a sprint. But unlike a marathon, the road any treatment must travel from the laboratory to ... Read more
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Amicus CEO John Crowley Outlines Progress in Batten, Pompe, Fabry Therapies
Pharmaceutical executives rarely make for a sympathetic Hollywood medical drama. But John Crowley did, ... -
Neurogene Opens Natural History Study of CLN7 and CLN5 Disease at UT Southwestern
Neurogene is sponsoring a natural history study to better understand the course of CLN5 ... -
Oklahoma Ranks Lowest on Programs Key to Rare Diseases on NORD’s 2019 State Report Card
Oklahoma suffers more tornadoes than any other state, has the highest per-capita rate of ... -
Rare Disease Groups Seek Public Support to Renew Newborn Screening Act in Senate
Screening newborns for genetic diseases with treatments that can prevent crippling or deadly progression, ... -
Amicus, Paragon to Collaborate on Gene Therapies for Batten, Other Diseases
Amicus Therapeutics entered a collaboration with Paragon Gene Therapy, a unit of Catalent Biologics, ...
North Carolina’s Indo Jax Surf Charities Helps Kids With Batten Disease, Other Special Needs
News
A small North Carolina nonprofit organization, Indo Jax Surf Charities, has recently gained attention for its set of free programs to help children with Batten disease and other special needs build self-esteem. Since launching these programs, Indo Jax has been featured ... Read more