00:00What is the role of phosphatase alkaline in hypophosphatase?
00:04Phosphatase alkaline does not work well in hypophosphatase,
00:08which is the fundamental enzyme to mineralize the skeleton,
00:12or to mineralize the dental apparatus.
00:15So, without this enzyme, we tend to have sick teeth,
00:19a sick skeleton, and fractures due to fragility,
00:22and also pseudo-fractures, a real osteomalacia.
00:26So, when we talk about a drug,
00:29we are talking about a substance, a molecule,
00:33that replaces the enzyme.
00:36So, we have a drug, which is an artificial, synthetic
00:40phosphatase alkaline, because it is produced
00:44in recombinant technology, which reaches the bone,
00:48and there it does what the enzyme, which has a mutated gene
00:52and therefore cannot do it efficiently,
00:55is now able to do it, so it replaces the missing enzyme.
01:00Italy did not have a great experience,
01:03simply because the drug was not refundable.
01:06So, we could only give it to the most serious patients,
01:10by making applications, which seemed like a grant application,
01:14to prepare a request for one of these drugs.
01:18I have had patients, and I have to say that there was
01:22a particular patient, who was now in bed,
01:25with femoral fractures, multiple vertebrae,
01:28assisted by the family.
01:31She had penile syndrome, she was going back and forth
01:35from the department of neurological urgency.
01:41She was very sick, full of pain, and was now in bed.
01:46After the pharmacological treatment, she came to my studio
01:50with only one stick, which was a miracle for me.
01:53She took a plane trip to come and see me.
01:56She got on the stairs of the plane, she got off,
02:01she came to me and said, look how well I am.
Comments