Neurodegenerative disease: Addressing unmet medical needs
_Parkinson's disease (PD)
PD is a a devastating neurodegenerative disease that is driven, for the most part by aging and, most likely tiggered, by environmental factors on a background of genetic susceptibility. It manifests itself through both motor and non-motor pathologies which, over time, progressively degrade the quality of life of the afflicted patient. Its incidence is particularly high in selected human populations (Ashkenazi Jews and North Africans) but affects all ethnic groups globally. It is currently estimated that there are about 6 million affected individuals worldwide. With the aging of the general population, the number of PD-afflicted patients is expected to double through 2030.
Motorneuron disease (‘MND’)
Also known as Amyotrophic lateral sclerosis (‘ALS’) is a relentless fatal paralytic disorder confined to the voluntary motor system. The onset of disease is usually in the fourth or fifth decade of life. The disease progresses rapidly, with a mean survival of 3 years (95% confidence interval, 2.6‑3.4 years) after diagnosis. Pathologically, ALS is characterized mainly by a loss of the upper motor neurons or the lower motor neurons or both. As a rare disease condition (prevalence of around 4-5 per 100,000 population) it presents in a genetic/familial form in 10% of the cases and is slightly more prevalent in males than in females (~60%). To date, only a few approved treatments, such as mechanical ventilation and riluzole, do prolong survival in ALS patients to a degree but this patient population is in desperate need of disease-modifying therapies to alleviate their suffering.
Multiple System Atrophy (MSA)
Multiple Systems Atrophy (MSA) is a sporadic adult-onset neurodegenerative disorder presenting four to five cases per 100,000 population; an orphan drug indication. Clinically it presents as a combination of motor and non-motor pathologies where the median life expectancy from onset is less than 9 years. There are no disease-modifying treatments and any current treatment is palliative, partially effective and transient at best. Confirmed diagnosis can only be made post mortem as the symptoms that these patients present can be indistinguishable from other neurodegenerative disorders such as Parkinson's disease. The need for diagnostic and therapeutic tools for MSA is urgent.
PD is a a devastating neurodegenerative disease that is driven, for the most part by aging and, most likely tiggered, by environmental factors on a background of genetic susceptibility. It manifests itself through both motor and non-motor pathologies which, over time, progressively degrade the quality of life of the afflicted patient. Its incidence is particularly high in selected human populations (Ashkenazi Jews and North Africans) but affects all ethnic groups globally. It is currently estimated that there are about 6 million affected individuals worldwide. With the aging of the general population, the number of PD-afflicted patients is expected to double through 2030.
Motorneuron disease (‘MND’)
Also known as Amyotrophic lateral sclerosis (‘ALS’) is a relentless fatal paralytic disorder confined to the voluntary motor system. The onset of disease is usually in the fourth or fifth decade of life. The disease progresses rapidly, with a mean survival of 3 years (95% confidence interval, 2.6‑3.4 years) after diagnosis. Pathologically, ALS is characterized mainly by a loss of the upper motor neurons or the lower motor neurons or both. As a rare disease condition (prevalence of around 4-5 per 100,000 population) it presents in a genetic/familial form in 10% of the cases and is slightly more prevalent in males than in females (~60%). To date, only a few approved treatments, such as mechanical ventilation and riluzole, do prolong survival in ALS patients to a degree but this patient population is in desperate need of disease-modifying therapies to alleviate their suffering.
Multiple System Atrophy (MSA)
Multiple Systems Atrophy (MSA) is a sporadic adult-onset neurodegenerative disorder presenting four to five cases per 100,000 population; an orphan drug indication. Clinically it presents as a combination of motor and non-motor pathologies where the median life expectancy from onset is less than 9 years. There are no disease-modifying treatments and any current treatment is palliative, partially effective and transient at best. Confirmed diagnosis can only be made post mortem as the symptoms that these patients present can be indistinguishable from other neurodegenerative disorders such as Parkinson's disease. The need for diagnostic and therapeutic tools for MSA is urgent.
