FXIII

Factor XIII Patients Get New Treatment

This is going to be quite a year with potentially new products.
Here’s one to start the year off right!
The FDA has approved Novo Nordisk’s Tretten® (coagulation factor XIII A-subunit [recombinant]) for routine prophylaxis of bleeding in people with congenital factor XIII (FXIII) A-subunit deficiency. This is one of
the rarest inherited bleeding disorders, with an incidence of 1 in 1 million to 1 in 5 million.
FXIII is composed of two subunits, A and B, with genes on two different chromosomes. FXIII deficiency is usually caused by a deficiency of the A-subunit. In the absence of FXIII, loosely formed clots are developed, leading to bleeding complications similar to those in severe hemophilia A. Tretten is the only recombinant treatment for the disorder
Why does this matter? Patients with congenital FXIII A-subunit deficiency have a lifelong susceptibility to bleeding, including intracranial hemorrhage (spontaneous bleeding into the brain), which could be life-threatening if untreated.
For more info: TRETTEN-US.com

Great Book I Just Read
Rebecca by Daphne du Maurier [Kindle]

Rebecca is a gothic romance/psychological thriller, much along the lines of Jane Eyre, that skillfully transports the reader
into the early 1900s: a young bride (never named) narrates the tale, about coming to the famed estate of Manderley, as the new bride of middle-aged Maxim de Winter, whose first wife drowned in a mysterious boating accident only a year ago. Rich in character, thought and detail, transfixed on the lush countryside, landscaped grounds and endless ocean as the story progresses. The mansion seems haunted by the first wife—Rebecca—and every act, every word by everyone, including Max, seems to scream how missed the charming and stunning Rebecca is, how hopelessly inadequate the unsophisticated, young new bride is, until the bride considers ending her life. But then …Things are not at all what they appear. Through subtle hints and plot twists, the real story emerges bit by bit to an amazing and gripping ending. The Hitchcock movie by the same name is also fantastic.  Four/five stars.

Lucky Number 13 part 2

Just last week I blogged about CSL Behring’s new factor XIII concentrate Corifact. Now I’ve learned that Novo Nordisk announced that a Biologic License Application (BLA) has been submitted to the US Food and Drug Administration (FDA) requesting the approval of a recombinant factor XIII compound for those with congenital Factor XIII (FXIII) deficiency.

Corifact is made from human blood plasma. This new drug is made from recombinant technology, which uses genetic engineering.

Factor XIII deficiency has a prevalence of one case per two million people, with an estimated 600 diagnosed patients worldwide, making it one of the rarest bleeding disorders.

A press release from Novo Nordisk states: “Positive results from a phase III trial examining the efficacy and safety of recombinant factor XIII for the prevention of bleeds associated with congenital FXIII deficiency showed that when compared to a historic control group of individuals who did not receive routine FXIII infusions, preventive treatment with monthly recombinant FXIII injections significantly decreased the number of bleeding episodes requiring treatment. These data were presented at the American Society of Haematology (ASH) meeting in December 2010, and marked the first completed phase III study conducted to study the use of a recombinant FXIII treatment to prevent bleeding episodes in congenital FXIII deficiency patients.”

It’s all great news for those who have factor XIII deficiency, and with the sad news from around the world, especially Japan, we need good news.

Good Book I Just Read

No One Here Gets Out Alive
Jerry Hopkins and Danny Sugerman

I’m continuing my reading streak on The Doors, the 1960s band that took America by storm. In this book, Jerry Hopkins (Rolling Stone) and Danny Sugerman (then teen office boy for Doors) profile the madness and magnificence of Jim Morrison, front man and silky-voice singer, whose depravity and self-destruction eventually upstaged the music itself and his three talented band members. The book details Morrison’s schizophrenic adolescence in which he, with an IQ of 150, devoured sophisticated philosophy and poetry books, and wrote poetry himself, but yet immaturely and cruelly harassed his younger brother, scared the elderly and openly mocked cripples. The first few chapters alone might end any hero worship of The Lizard King. The book recounts chronologically how the Doors formed, their skyrocketing rise to fame, and the toll of fame on the unstable Morrison. They were only 21-23 years old, in a music industry and culture awash in drugs, but why did only Morrison succumb to the pressure? The book is insightful into the music industry of the 1960s. It’s incomplete in that no other character is truly explored: Manzarek, Densmore and Krieger are barely mentioned. They were together for six intense years: how did they cope with Morrison’s descent into madness? The book also sheds little insight into Morrison’s state of mind, or why he acts as he does other than the drugs or alcohol, but factually states a portrait of a seeming sociopath: the dozens of paternity suits, his wanting his child to be aborted because he simply didn’t want one, his cruelty to his mother when he performed, his promiscuity and debauchery.

It’s hard to reconcile the deep thinker with the raging drunk. Perhaps the book doesn’t go deep enough into Morrison’s psyche, but then, Morrison didn’t seem to let anyone in. He internalizes his pain, which seeps out in frightening rages, and then is dampened by alcohol. One only needs to look at his photo progression in just six years: from sleek, sexy rock star to bloated, bearded drunk. I don’t think the pain was from being a frustrated poet or even a rock star; that’s putting the cart before the horse. His pain was chronic, malignant. Poetry was one expression; rage and cruelty was another. Adoration from the masses was one treatment; alcohol, maybe heroin, was another, and became the final exit.

Morrison helped put the Doors on the map, and he destroyed the Doors by destroying himself. Forty years later, it seems we will never know what drove Morrison to the edge, and then over. The authors skim over any analysis by comparing his angst to being like the Greek god Dionysus, or part of the Beat generation, or expressing himself like the Indian shamans he revered. But this is shallow. Looking back today, Morrison was an emotionally disturbed artist who sought to medicate his pain through alcohol, and expressed himself through rage. Incredibly, he left a legacy of beautiful, mystical music that captured a unique time in America, and the black hole of his inner life. And we are drawn to such people, scared of them, and yet worship them. Despite the incomplete picture the book paints, it was a great read and I could not put it down. Three stars.

Lucky Number 13


When is the number 13 lucky?

We now have a factor concentrate for people with factor XIII deficiency. CSL Behring, makers of Helixate FS, Monoclate P and Mononine, was granted FDA approval for their factor XIII concentrate Corifact, indicated to treat congenital factor XIII deficiency. This is the first and only U.S. approved treatment for factor XIII-deficiency and the fourth new product that CSL Behring has brought to the U.S. market in the past two years. Corifact is already available for use in 12 countries throughout the world under the trade name Fibrogammin- P.

Factor XIII deficiency, also known as fibrin-stabilizing factor deficiency, is rare, affecting one in two million, with an incidence in the U.S. of approximately 150 people.

Symptoms include bleeding from the umbilical cord after birth, poor wound healing, miscarriages, subcutaneous bleeding, and excessive bleeding in joints and muscles following trauma. Patients lacking the FXIII protein are also at high-risk for intracranial hemorrhage (ICH), bleeding inside the skull that can be life threatening. Studies have shown that between 25- 60% of factor XIII deficient patients will experience an ICH at least once during their lifetime.

This is great news for those with factor XIII deficiency. For more information, please see www.corifact.com and contact your HTC staff.


Great Book I Just Read
Riders on the Storm by John Densmore

“There’s the Beatles, the Stones, and the Doors,” says Paul Rothschild, producer of one of the greatest bands of the sixties. Drummer John Densmore was only 21 when he joined with three other California students to form a band that would soon skyrocket to the top at a time when the US was a nation at war in Vietnam, and with itself. With quiet Robby Krieger on guitar, methodical and rational Ray Manzarek on keyboard and Adonis-look-a-like singer Jim Morrison, the band was like no other. With no bass player, they combined blues, jazz and psychedelic rock, with some Indian strings and often very dark lyrics, about death, murder and rage. And no wonder: Not long after forming, Densmore writes about their iconic lead singer, “I’m in a band with a psychotic.”

The book is captivating. It starts in Paris, 1975, with Densmore visiting Morrison’s grave, four years after his death at age 27 on July 3, 1971 from an overdose. Returning to the hotel, he pours his feelings out in a “letter” to Morrison on the hotel stationery, which then gets interspersed throughout the book with his recollections. His memoir details his time in the band, returning now and then to the ongoing letter, and towards the end, “updates” Morrison on events of the 1980s and 1990s. Interspersed are appropriate lyrics from their songs, so that the book itself becomes quite artsy. The book gives excellent insight into what the 60s were like, what it’s like being in a band on the rise and what it was like to survive the onslaught of Jim Morrison. What’s missing, though, is what “The Lizard King” was really like. We get snapshots, snippets, and stories, but it’s as if Densmore viewed Morrison at arm’s length, even though he intensely shared six years with him. He was in awe of Morrison—who wasn’t?—and scared of him, and rightly so. Though he feels guilty at not being able to help Morrison, we have to remember he was only a young man from a humble Catholic background, ill equipped to cope with sudden stardom, wealth and the phenomenally complex, creative and self-destructive Morrison.

As if to highlight the detachment Densmore had with Morrison, Densmore dedicates the book to John Lennon of the Beatles, one of Densmore’s heroes, and also mentions Lennon’s assassination in his “letter” to Morrison, yet doesn’t mention or maybe doesn’t even know that Lennon was assassinated on Morrison’s birthday, a fact he should know as Morrison and Densmore were born only a week apart.

“We sensed rage and a possible explosion too near the surface to mess with in dealing with you,” Densmore writes in his letter to the now dead Morrison. “It seemed to have a lid on it—Pandora’s box with all the demons that wanted to be released. We never opened the box… we had to deal with your demons seeping out the side.” They seeped out and poisoned everything; the Doors had to cancel a 20-city tour when Morrison was arrested in Miami for indecent exposure at a concert (he was just pardoned this past December!). The charisma, lyrics and stunning voice of Morrison helped make the Doors a success; his addiction, rage and irresponsibility destroyed them. The book was published in 1990 and Densmore writes, “Well, we’re going on 20 years and there’s no end in sight” of the fascination people have with the Doors. On July 3, it will be 40 years since Morrison’s death, and the Doors are almost as popular as ever. I picked this book up and finished it 5 hours later—it’s compelling, honest, shocking and eye opening. A great book about one of my favorite bands and my favorite singer—for no one could sing like Jim Morrison. Three stars.

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