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Cipaglucosidase alfa, sold under the brand name Pombiliti, and used in combination with miglustat, is a medication used for the treatment of glycogen storage disease type II (Pompe disease). [ 4 ] [ 5 ] Cipaglucosidase alfa is a recombinant human acid α-glucosidase enzyme replacement therapy that provides an exogenous source of acid α ...
In September 2023, the FDA approved a two-component therapy of Pombiliti (cipaglucosidase alfa-atga) and Opfolda 65 mg capsules for adults living with late-onset Pompe disease weighing more than 40 kg and who are not improving on their current enzyme replacement therapy. [19] [20]
Miglustat is indicated to treat adults with mild to moderate type I Gaucher disease for whom enzyme replacement therapy is unsuitable. [14]In the European Union, miglustat (Opfolda), in combination with cipaglucosidase alfa, is a long-term enzyme replacement therapy in adults with late-onset Pompe disease (acid α‑glucosidase [GAA] deficiency).
This multi-page article lists pharmaceutical drugs alphabetically by name. Many drugs have more than one name and, therefore, the same drug may be listed more than once. ...
Notation (,)Parameters {,,, …} – number of trials[,] – success probability for each trial= Support {,, …,} – number of successesPMF ()CDF ...
In probability theory and statistics, a normal distribution or Gaussian distribution is a type of continuous probability distribution for a real-valued random variable.The general form of its probability density function is [2] [3] = ().
Probability and gambling have been ideas since long before the invention of poker. The development of probability theory in the late 1400s was attributed to gambling; when playing a game with high stakes, players wanted to know what the chance of winning would be.
Box plot and probability density function of a normal distribution N(0, σ 2). Geometric visualisation of the mode, median and mean of an arbitrary unimodal probability density function.