Thelansis

 Cerebral adrenoleukodystrophy (CALD) is the most severe form of adrenoleukodystrophy (ALD), a rare metabolic disorder linked to the X chromosome. Typically occurring between ages 3 and 12, around 40% to 72% of boys diagnosed with ALD eventually progress to cerebral ALD. This condition leads to a rapid and severe decline in neurological function, often resulting in death for untreated patients. For affected boys and their families, cerebral ALD is a devastating affliction, marked by a profound symptom burden and a swift deterioration in neurological function when not treated. Of particular clinical significance are the six major functional disabilities associated with cerebral ALD, as they can significantly impede a patient's ability to function independently. These six disabilities encompass loss of communication, cortical blindness, reliance on tube feeding, complete incontinence, dependence on a wheelchair, and the complete loss of voluntary movement. ·    ...

Cerebral adrenoleukodystrophy (CALD) is the most severe manifestation of adrenoleukodystrophy (ALD), a rare, X-linked metabolic disorder. Approximately 40% to 72% of boys diagnosed with ALD will progress to cerebral ALD, typically between the ages of 3 and 12. Cerebral ALD is characterized by a rapidly progressive neurologic decline leading to a severe loss of neurologic function and death in most untreated patients. It is a devastating condition for affected boys and their families, with a profound symptom burden and rapid decline in neurologic function without treatment. Cerebral ALD is associated with six major functional disabilities; they are of particular clinical importance because they can significantly compromise a patient’s ability to function independently. The six MFDs are loss of communication, cortical blindness, tube feeding, total incontinence, wheelchair dependence, and complete loss of voluntary movement. It is important to put together a care team as early as possi...

  A progressive peroxisomal disease, characterized by endocrine dysfunction, progressive myelopathy, and peripheral neuropathy, and leukodystrophy. Age of onset is highly variable, but often in the first decade. In 30–40% of boys with ALD, a CNS-based neuroinflammatory process is initiated between the ages of 4–10 years known as cerebral ALD (cALD).   Epidemiology- Based on literature research and epidemiology study of Thelansis, the diagnosed prevalence of Cerebral Adrenoleukodystrophy (CALD) are approximate ~15,600 cases in 8 major markets for the year 2020.   The competitive landscape of Cerebral adrenoleukodystrophy (CALD) includes country-specific approved as well as pipeline therapies. Any asset/product-specific designation or review and Accelerated Approval are being tracked and supplemented with analyst commentary. KOLs insights of Cerebral adrenoleukodystrophy (CALD) across 8 MM market from the center of Excellence/ Public/ Private hospitals particip...