Title: Desperate Plea for Life-Saving Drug Ryplazim as Supplies Vanish
Subtitle: 14-month-old baby’s life hangs in the balance due to unavailability of treatment
Date: [Insert Date]
Byline: [Insert Author Name]
[Lecce, Italy] – The life of a 14-month-old baby named Nicolò is at risk as the drug Ryplazim, crucial for treating his rare condition, plasminogen deficiency, has become unobtainable. Ryplazim, manufactured by Kedrion, is the first FDA-approved pharmacological treatment for this ultra-rare pathology. However, bureaucratic problems have led to the suspension of the drug, leaving Nicolò’s life hanging in the balance.
Federica Parata, Nicolò’s mother, has resorted to launching an appeal to international institutions through a heartfelt letter posted on the web and sent via certified emails. In her emotional plea, Parata describes the devastating challenges Nicolò has faced since birth, including hydrocephalus, Dandy Walker syndrome, and the emergence of plasminogen deficiency. The young mother stresses the urgency of finding Ryplazim to save her son’s life.
Nicolò’s medical journey has forced the Parata family into a difficult situation. While Parata resides in Lecce with her mother for additional caregiving support, her partner Alessandro, who works in Verona, travels between hospitals in Lecce and Rome where Nicolò has spent most of his life. Amid this tumultuous situation, Parata emphasizes the importance of finding a solution to save Nicolò’s life, citing their rightful access to healthcare as guaranteed by Italian law.
The absence of Ryplazim poses a significant obstacle to Nicolò’s treatment. He requires three weekly plasma infusions, a non-resolving therapy that has also been discontinued in the United States due to the unavailability of Ryplazim. Consequently, transferral to the United States for treatment is not a viable option, given the drug’s scarcity in Italy and Europe. In a stroke of fortune, Nicolò has received limited doses of Ryplazim for compassionate use, thanks to the involvement of the Italian Federation of Rare Diseases onlus and Gemelli doctors.
Despite the recent arrival of eight bottles of Ryplazim, which will cover a limited period, Nicolò’s life remains in constant jeopardy. He recently underwent yet another surgery, necessitated by the implantation of a drainage system in his head. However, external catheters used in the procedure are prone to infections, increasing the risks associated with plasma transfusions. The upcoming operation requires adequate Ryplazim coverage to ensure his safety.
The uncertainty surrounding Nicolò’s life expectancy looms large. While some patients with plasminogen deficiency lead fairly normal lives for up to 50 years with treatment, the absence of Ryplazim leaves an indefinite timeline. Fibrin membranes formed due to the deficit have already obstructed shunts at the abdominal level, necessitating Nicolò’s upcoming abdominal cleaning procedure for membrane removal and catheter repositioning.
A bottle of Ryplazim costs €2,000, a seemingly insignificant amount compared to the Puglia Region’s daily expenditure of €1,400 to provide eye drops for Nicolò. As desperation mounts, Parata has appealed to political institutions for the initiation of a European clinical trial. She has also suggested the production of Ryplazim by the Italian military pharmacy, but was informed that it can only produce listed drugs, excluding the life-saving treatment.
Despite the recent small success Nicolò experienced while on continuous Ryplazim treatment, increasing his plasminogen activity to 32 percent, his future remains uncertain without access to the systemic drug.
As the young family faces this uphill battle against time, they implore any person or institution who can intervene to help secure Ryplazim and provide Nicolò with a fighting chance at life.
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