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Browse Rare Disease Advisor’s engaging “Rare Care” podcast series, featuring interviews with leading researchers, stakeholders, and influencers within the rare disease community.

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Mental Health Toll Reported in Patients With Myasthenia Gravis

“Although rare conditions by definition affect no more than 1 in 2000 individuals, there are over 6000 known conditions so that collectively they represent a substantial health burden, with an estimated 3.5 million affected individuals in the UK,” Spencer-Tansley and colleagues wrote in BMC Health Services Research.  The UK population is around 67 million; this…

Doctor and patient talking

Understanding Hereditary Angioedema and Associated Comorbidities

While we as clinicians often excel at focusing on the primary diagnosis, especially in the case of a rare disorder, it is important that we also treat all other comorbidities with care; this is because we know that comorbidities are often aggravated by the primary diagnosis and vice versa.  This becomes more urgent as patients…

Clinical trial imagery

Assessing Existing NMOSD Therapeutic Strategies

Neuromyelitis optica spectrum disorder (NMOSD) is characterized by relapsing attacks, driven by inflammation of the central nervous system. This typically manifests as either optic neuritis or longitudinally extensive transverse myelitis.  While some patients are known to have a monophasic disease course, they are decidedly in the minority; most patients with NMOSD experience devastating relapses that…

NMOSD differential diagnosis

Tackling Misdiagnosis in NMOSD

In medicine, you cannot treat what you do not diagnose. An undiagnosed/misdiagnosed condition can lead to unnecessary tests and ineffective treatment: an unworkable set of circumstances that leads to nowhere.  How common is it for diseases to be misdiagnosed? When I was a medical student, I was informed about the case of a patient with…

Occupational therapy

Assessing the Role of Occupational Therapy in hATTR

Patients with hereditary transthyretin amyloidosis (hATTR) have to contend with a range of physical and psychological symptoms that can be debilitating. Because amyloid deposition occurs throughout the body, patients are subject to multisystemic symptoms; among these are progressive polyneuropathy, which first manifests as mild autonomic dysfunction and lower limb sensory impairment and progresses to more…

Human stem cells

The Reparative Mechanisms of the Lungs and the Future of IPF

Despite the ubiquitous threat of diseases and accidents, the human body retains a wonderful ability to repair and regenerate itself. For this, credit is primarily due to the immune system; without it, human beings would simply die from the smallest of wounds. In addition, thrombostatic agents ensure that we stop bleeding from a cut in…

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