Perhaps 70- 85% of rare diseases have a root in genetics, - which is why continuous research and reporting is so critical for those with rare conditions. This is exactly…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
Most people have probably heard the saying, “change your thoughts, change your life.” There is great truth to this because how we think can significantly impact our mental and physical…
Pulmonary arterial hypertension (PAH), a rare and chronic disorder characterized by high blood pressure (hypertension) in the lungs that causes the pulmonary arteries to thicken or harden, most often manifests…
Rare Community Profiles is a Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their families, advocates, scientists, and more.…
Neurological conditions now have the highest rates of disability and ill health worldwide. The Disparity Although more than 80% of the neurological deaths and nervous system disorders occur in lower…
Pulmonary arterial hypertension (PAH) is a rare, chronic, and progressive disorder characterized by high blood pressure in the lungs. This causes pulmonary arteries to thicken or harden, which eventually puts…
Rare Community Profiles is a Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their families, advocates, scientists, and more.…
Jenny Decker began her solo voyage in her 1983 Bristol 35.5 Tiama from Honokōhau, Hawaii on June 28, 2023 (see Patient Worthy articles Part 1 and Part 2.) Jenny hopes…
Rare Community Profiles is a Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their families, advocates, scientists, and more.…
Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…
A study by a team of scientists at the Children’s Research Institute was reported this week in the journal Nature Communications. The study is welcomed by the medical profession as…
Historically, it has been difficult to incentivize companies to develop therapies for rare or “orphan” conditions. The National Conference of State Legislatures (NCSL) explains that “orphan” conditions are: neglected conditions…
There are limited therapeutic interventions for those living with Niemann-Pick disease type C (NPC), a rare and progressive genetic disorder. Unfortunately, many people with NPC experience severe and life-threatening complications…
The Duchenne muscular dystrophy (DMD) community has a reason to celebrate. According to a recent news release, the FDA recently approved Duvyzat (givinostat) for people ages six and older who…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
Ronald Acuña Jr. is a professional baseball player with the Atlanta Braves. The powerhouse player is known for being an NL MVP and for becoming the first player in Major…
There are over 30 subtypes of congenital muscular dystrophy (CMD), or muscular dystrophies that are present at birth. These rare genetic disorders often manifest in hypotonia (low muscle tone) and…
Rare Community Profiles is a Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their families, advocates, scientists, and more.…
People with Cushing's disease have a benign pituitary tumor or pituitary growth that, when triggered, overproduced adrenocorticotropic hormone (ACTH). Too much ACTH causes the body to also overproduce cortisol. Known as…
Dupixent has long been known as a therapy which reduces itch, slows disease progression, and reduces exacerbations in a number of allergic conditions such as asthma, eczema, and nasal polyps.…
Rare Community Profiles is a Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their families, advocates, scientists, and more.…
Parkinson's disease is a progressive neurodegenerative disease. As neurons in the brain degenerate and die, those affected experience symptoms such as tremors or shaking in the hands, slowed movement, muscle…
Right now, no cure exists for people living with systemic sclerosis (also known as systemic scleroderma). There are treatment options available to manage systems. However, these also come with issues.…
Rare Community Profiles is a Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their families, advocates, scientists, and more.…
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