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Presymptomatic patients can be cured with one treatment of atidarsagene autotemcel, which is a type of advanced medicine called a ‘gene therapy’. This type of medicine works by delivering genes into the body. The active substance in atidarsagene autotemcel is CD34+ stem cells. They are retrieved from the patient's own bone marrow or blood.
The Merck Manuals (outside the U.S. and Canada: The MSD Manuals; Chinese: 默沙东诊疗手册; pinyin: Mòshādōng Zhěnliáo Shǒucè) are medical references published by the American pharmaceutical company Merck & Co. (known as MSD outside the United States and Canada), that cover a wide range of medical topics, including disorders, tests, diagnoses, and drugs.
Multiple sulfatase deficiency (MSD), also known as Austin disease, [1] or mucosulfatidosis, [1] is a very rare autosomal recessive [2] lysosomal storage disease [3] caused by a deficiency in multiple sulfatase enzymes, or in formylglycine-generating enzyme, which activates sulfatases.
Duchenne muscular dystrophy, or DMD, is a muscle-wasting disease caused by a genetic mutation in the dystrophin gene -- the largest gene on the X chromosome -- which affects one in every 2,400 ...
In the United States, the Doctor of Dental Surgery and Doctor of Dental Medicine are terminal professional doctorates, which qualify a professional for licensure. The DDS and DMD degrees are considered equivalent. The American Dental Association specifies: The DDS (Doctor of Dental Surgery) and DMD (Doctor of Dental Medicine) are the same degrees.
The main discussion of these abbreviations in the context of drug prescriptions and other medical prescriptions is at List of abbreviations used in medical prescriptions. Some of these abbreviations are best not used, as marked and explained here.
Neuromuscular medicine: Symptoms: Increasing weakening, breakdown of skeletal muscles, trouble walking [1] [2] Duration: Chronic [1] Types > 30, including Duchenne muscular dystrophy, Becker muscular dystrophy, facioscapulohumeral muscular dystrophy, limb–girdle muscular dystrophy, myotonic dystrophy [1] [2] Causes
Other pre-existing bone-marrow disorders such as acquired aplastic anemia following immunosuppressive treatment and Fanconi anemia can evolve into MDS. [ 15 ] MDS is thought to arise from mutations in the multipotent bone-marrow stem cell , but the specific defects responsible for these diseases remain poorly understood.
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