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  Nido Biosciences, a biopharmaceutical company developing medicines to treat debilitating neurological diseases with precision, today announced that the European Medicines Agency has granted an Orphan Drug Designation for its lead clinical candidate, NIDO-361. This novel small molecule is designed to treat the spinal and bulbar muscular atrophy, also known as Kennedy’s disease; the designation has been made after the company reported full enrollment of the Phase 2 study for the medicine. Publish Date: 22-10-2024   Source: Nido Biosciences Kennedy’s disease, called bulbospinal muscular atrophy (BSMA), is a rare genetic disorder following an X-linked recessive inheritance pattern characterized by the degeneration of motor neurons leading to muscle wasting in the proximal and bulbar regions. BSMA is caused by an unstable expansion of a CAG triplet repeat (40-62 repeats) in exon 1 of the androgen receptor (AR) gene located on chromosome Xq11-12. The onset of symptoms typically oc

  Kennedy's disease, called bulbospinal muscular atrophy (BSMA), is a rare genetic disorder following an X-linked recessive inheritance pattern characterized by the degeneration of motor neurons leading to muscle wasting in the proximal and bulbar regions. BSMA is caused by an unstable expansion of a CAG triplet repeat (40-62 repeats) in exon 1 of the androgen receptor (AR) gene located on chromosome Xq11-12. The onset of symptoms typically occurs between the ages of 30 and 60 years, and initial clinical manifestations may include tremors, muscle cramps, twitching, fatigue, and slurred speech. With disease progression, patients may experience weakness and wasting of the limb and bulbar muscles, resulting in dysarthria, dysphonia, hanging jaw, tongue wasting, chewing difficulty, and impaired mobility. The disease may be misdiagnosed initially as hereditary spastic paraplegia, spinocerebellar ataxia, other motor neuron diseases, myopathies, neuropathies, or cervical spondylosis. Th

Kennedy's disease, called bulbospinal muscular atrophy (BSMA), is a rare genetic disorder following an X-linked recessive inheritance pattern characterized by the degeneration of motor neurons leading to muscle wasting in the proximal and bulbar regions. BSMA is caused by an unstable expansion of a CAG triplet repeat (40-62 repeats) in exon 1 of the androgen receptor (AR) gene located on chromosome Xq11-12. The onset of symptoms typically occurs between the ages of 30 and 60 years, and initial clinical manifestations may include tremors, muscle cramps, twitching, fatigue, and slurred speech. With disease progression, patients may experience weakness and wasting of the limb and bulbar muscles, resulting in dysarthria, dysphonia, hanging jaw, tongue wasting, chewing difficulty, and impaired mobility. The disease may be misdiagnosed initially as hereditary spastic paraplegia, spinocerebellar ataxia, other motor neuron diseases, myopathies, neuropathies, or cervical spondylosis. The pr