A Global Alliance to Save Lives: How Two Parents and PackGene Are Redefining What's Possible in Gene Therapy with Help from Weill Cornell Medicine
A Global Alliance to Save Lives58 How Two Parents and PackGene Are Redefining Whats Possible in Gene Therapy with Help from ...
BlackfinBio Ltd announces the acquisition of Parkinson's IP patent portfolio from OXB
BlackfinBio Ltd (BFB) a clinical stage gene therapy company focused on the development of treatments for rare neurological diseases has announced today that it has acquired the Parkinson's patent ...
Daily Mail26d
My baby girl was hitting all her milestones - then, on her first birthday, a vanishingly rare disease took everything from her. A cure will cost millions, but we'll never stop ...
We had every gene in her body mapped, and at 14 months old, Tallulah was officially diagnosed with Hereditary Spastic Paraplegia Type 56 (SPG56) - an incurable brain disease so rare no one else in ...
BMJ13d
Bladder dysfunction in hereditary spastic paraplegia: what to expect?
kliinikum.ee Background Hereditary spastic paraplegia (HSP) comprises a group of rare neurodegenerative disorders characterised by progressive spasticity and hyperreflexia of the legs. Neurogenic ...
casereports.bmj21d
Identification of a novel MAG gene mutation with 22q11.21 microduplication linked to hereditary spastic paraplegia
Diagnosing hereditary spastic paraplegia (HSP) in paediatric patients can be challenging, especially when there is no positive family history. Children are often initially misdiagnosed with cerebral ...