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3 days ago ˇ Please note that this is just a selection of Orphanet's rare disease epidemiological data. ... atrophy-microcephaly-muscle weakness- optic atrophy syndrome.
6 days ago ˇ A rare disease therapy as such can be defined as a treatment that is intended to heal or relieve one or more of the 6000-8000 rare disorders. These therapies ...
5 days ago ˇ What Is a Rare Disease? “Rare disorders are defined as less than 200,000 affected patients in the U.S.,” says Dr. Jerry Vockley, Chief of Medical Genetics ...
5 days ago ˇ Atrophy: Thinning muscles and loss of strength. Fasciculations: A spontaneous and involuntary contraction of muscles that may be visible as twitching under ...
4 days ago ˇ Progeroid syndromes are a group of diseases that cause individuals to age faster than usual, leading to them appearing older than they actually are. People born ...
6 days ago ˇ SCA, a group of rare genetic disorders, causes progressively debilitating neurological symptoms in patients including clumsiness, muscle weakness, and tremors.
10 hours ago ˇ The World Orphan Drug Congress Europe is the largest orphan drug and rare disease event across the globe.
6 days ago ˇ It is a negative regulator of glucose homeostasis and is involved in energy metabolism, inflammation, ER-stress, mitochondrial dysfunction, and apoptotic ...
2 days ago ˇ Haploinsufficiency is caused a heterozygous loss-of-function gene mutation. ˇ Because of the presence of a normal allele, haploinsufficiency is a unique group of ...
3 days ago ˇ CWD is a fatal neurological disease that affects white-tailed deer. It is a member of a group of diseases called transmissible spongiform encephalopathies, ...
Missing: atrophy | Show results with:atrophy
