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Changing My Perspective on a Rare Disease
Where were you on Feb. 28? Did you know that it was Rare Disease Day? Don’t feel bad if you missed it because I did too. But I shouldn’t have. Because Rare Disease Day, along with the international events around it, represented a major change in perspective toward finding…
ACT Support Program Empowers Caregivers to Cope With Distress, Study Says
Caregivers of people with amyotrophic lateral sclerosis (ALS) and progressive muscular atrophy (PMA) say a Dutch support program — one based on Acceptance and Commitment Therapy — empowered them to make better choices in line with their own needs, and to cope with distress, a study shows. The…
‘Rare Barometer’ Program Helps Eurordis Shape EU Rare Disease Policy
People with rare diseases know that the right government policies can make a big difference in the quality of their own lives, and those of their caregivers. But most lawmakers aren’t experts in even one well-known disease — let alone the world’s estimated 7,000 rare disorders. So how does the…
Scientists Use Patient Cells, Chip Technology to Re-create Blood-Brain Barrier Defects
Researchers at California’s Cedars-Sinai Medical Center have re-created the blood-brain barrier, a vital component of the central nervous system, using Organ-Chip technology by Emulate. This advances the possibility of patient-specific treatments for neurodegenerative disorders, including amyotrophic lateral sclerosis (ALS). Blood-brain barrier defect has been linked to…
AB Science’s New Masitinib Study Validated by EMA, With Expected Start Later This Year, Company Says
AB Science has optimized the design of a Phase 3 confirmatory study for mastinib, its oral treatment for amyotrophic lateral sclerosis (ALS), to avoid any possibility of bias. The optimized protocol for the study (NCT03127267), not yet recruiting, was validated by the European Medicines Agency (EMA). Although AB…
Muscular Symptoms
Amyotrophic lateral sclerosis (ALS) is a neurological disorder that is characterized by the progressive loss of motor neurons or nerve cells that control voluntary muscle movements. One prominent problem for ALS patients is muscle weakness. This affects their ability to perform everyday tasks, such as walking, speaking, and eating.
Digestive Symptoms
Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease characterized by the death of motor neurons — the nerve cells that control muscle movement. The primary symptoms of ALS include muscle weakness, tremors, and cramps. However, patients may experience other symptoms — including digestive problems that may contribute…
Anxiety and Depression
Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disorder characterized by the progressive loss of motor nerves, which are the nerve cells that control muscle movement. As the disease progresses, patients develop disabilities that worsen over time. Within a few years of diagnosis, patients may lose the ability to…
Anti-Inflammatory/Neuroprotective Agents
Amyotrophic lateral sclerosis (ALS) is a progressive disease characterized by the loss of motor neurons, the nerve cells that control muscle movement. As the disease progresses, nerve cells die, leading to muscle weakness and other symptoms of the disease. A factor that contributes to disease progression is an increase…