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Latest articles published - Methylmalonic acidemia

Living with a rare disease: An often underestimated psychological challenge

Rare diseases

Living with a rare disease: An often underestimated psychological challenge

Feb 28, 2025 • 2 Likes

What is a rare disease? A rare disease is a condition that affects a limited number of people in a given population. There is no universal definition of rare diseases. In fact, prevalence can be...

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What are the clinical research avenues in certain rare diseases?

Rare diseases

What are the clinical research avenues in certain rare diseases?

Dec 24, 2021

This article is not about listing all the drug candidates that are currently being tested, for six diseases mentioned above. Research is ongoing, and a lot of treatments are the subject of clinical...

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Lysosomal storage disorders: A care journey with many challenges

Rare diseases

Lysosomal storage disorders: A care journey with many challenges

Sep 25, 2021

What are lysosomal storage disorders?Lysosomal storage disorders (LSDs) are a group of about fifty, rare and inherited metabolic diseases. These are genetic diseases that impact cell function and...

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Rare Disease Day: Isolation and psychological impact, let's talk about it!

Rare diseases

Rare Disease Day: Isolation and psychological impact, let's talk about it!

Feb 28, 2021

Rare diseases: diverse and complex pathologiesFor a condition to be classified as a rare disease, it must affect less than one person in 2,000. These diseases will affect the patient's life in a...

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Acromegaly, Cushing's disease... Learn more about pituitary neuroendocrine tumors!

Rare diseases

Acromegaly, Cushing's disease... Learn more about pituitary neuroendocrine tumors!

Aug 6, 2020 • 1 comment

What is the pituitary gland? To fully understand what pituitary neuroendocrine tumors (pitNET) are, we first need to understand what the pituitary gland is and its role. Also called the hypophysis,...

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US Approves First RNA Interference Drug

Methylmalonic acidemia

US Approves First RNA Interference Drug

Sep 4, 2018 • 1 comment

Alnylam’s Onpattro (patisiran) can treat a rare hereditary disease by silencing a specific gene. The first therapy based on RNA interference (RNAi) gene silencing has been approved by the US Food and...

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