Van beleggers
voor beleggers
Uitgebreid zoeken

Beurscodes, betekenis en hulp bij zoeken

Europa

AEX
Euronext Amsterdam
BRU
Euronext Brussels
PSE
Euronext Paris
LIS
Euronext Lissabon
CHX
CBOE Europe, grote(re) EU aandelen
NAV
Investment Funds (NAV)

Noord-Amerika

NYS
New York Stock Exchange
OTC
CBOE BZX Exchange (US)
TSE
Toronto Stock Exchange

Kunt u een instrument niet vinden?

Zoek dan via de zogenaamde ISIN code. Elk instrument, aandeel etc. heeft een unieke code.

Kies vervolgens - wanneer er meerdere resultaten zijn - de notering op de beurs van uw keuze.

Waar vind ik die ISIN code?

Google de naam van het instrument, aandeel etc. met de toevoeging 'ISIN'.

Als zoeken op ISIN code geen resultaten oplevert hebben wij het instrument of aandeel niet in onze koersendatabase.

desktop iconMarkt Monitor
  • Word abonnee
  • Inloggen

    Inloggen

    • Geen account? Registreren

    Wachtwoord vergeten?
Home  /  Forum  /  Pharming  /  F.A.Q,s betreffende HAE

Ontvang nu dagelijks onze kooptips!

word abonnee

Aandeel Pharming Group AEX:PHARM.NL, NL0010391025

  • 0,845 17 mei 2024 17:35
  • -0,014 (-1,63%) Dagrange 0,845 - 0,864
  • 3.070.825 Gem. (3M) 6,5M

F.A.Q,s betreffende HAE

11 Posts
| Omlaag ↓
  1. [verwijderd] 16 november 2003 21:34
    Waarschijnlijk al eerdThe Hereditary Angioedema Association
    presents
    Frequently Asked Questions about Hereditary Angioedema

    What is Hereditary Angioedema?

    Hereditary Angioedema (HAE) is a rare and serious genetic condition occurring in about 1/10,000 to 1/50,000 individuals. The disease is characterized by episodes of edema (swelling) in body parts, most notably the hands, feet, face, and airway passages. In addition, patients often have bouts of excruciating abdominal pain, nausea, and vomiting that is caused by swelling in the intestinal wall.

    HAE patients have a defect in the gene that controls a blood protein called C1-inhibitor. The genetic defect results in production of either inadequate or nonfunctioning C1-inhibitor protein. Normal C1-inhibitor helps to regulate the complex biochemical interactions of blood based systems involved in disease fighting, inflammatory response, and coagulation. Because defective C1-inhibitor does not adequately perform its regulatory function, a biochemical imbalance can occur and produce unwanted peptides that induce the capillaries to release fluids into surrounding tissues, thereby causing edema.

    HAE is called hereditary because the genetic defect is passed on in families. A child has a 50 percent chance of inheriting this disease if one of his or her parents has it. The absence of family history does not rule out the HAE diagnosis, however. Scientists report that as many as 20 percent of HAE cases result from patients who had a spontaneous mutation of the C1-inhibitor gene at conception. These patients can pass the defective gene to their offspring.

    Because the disease is very rare, it is not uncommon for patients to remain undiagnosed for many years. Many patients report that their frequent and severe abdominal pain was inappropriately diagnosed as psychosomatic, resulting in referral for psychiatric evaluation. Unnecessary exploratory surgery has been performed on patients experiencing gastrointestinal edema because abdominal HAE attacks mimic a surgical abdomen. Before therapy became available, the mortality rate from airway obstruction was reportedly as high as 30%.

    er geplaatst, maar basiskennis voor newbees:
  2. [verwijderd] 16 november 2003 21:35
    What causes Hereditary Angioedema attacks?

    Most attacks occur spontaneously with no apparent reason, however anxiety, stress, minor trauma, surgery, and illnesses such as colds and flu have been cited as triggers. Dental procedures make HAE patients particularly vulnerable to airway attacks. Patients have also reported swelling in extremities following typing, prolonged writing, pushing a lawn mower, hammering, shoveling, and other physical activities.

    In women, menstruation and pregnancy seem to have a major effect on disease activity. Some women patients report a definite increase in the number of attacks during their menstrual periods. During pregnancy, some patients note an increase in the frequency of attacks, while others have reported a decrease. Use of oral contraceptives is associated with an increase in the frequency and severity of attacks.

  3. [verwijderd] 16 november 2003 21:36
    How is Hereditary Angioedema diagnosed?

    Most cases of angioedema are not HAE because they are caused by something other than C1-inhibitor deficiency. Laboratory analysis or genetic testing must confirm the HAE diagnosis. There are two specific blood tests that confirm HAE:

    1). C1-inhibitor quantitative (antigenic)
    2). C1-inhibitor functional

    Traditionally, the disease has been classified into two types. The most common form of the disease--Type I--is characterized by low quantitative levels of C1-inhibitor and affects about 85% of patients. Type II HAE affects the other 15% and is characterized by normal or elevated levels of C1-inhibitor that has abnormal function. A paper in the September 2000 volume of the British medical journal Lancet suggests there is another form of HAE that only affects females in families, but is not caused by C1-inhibitor deficiency. It has been proposed that this disease be labeled HAE Type III.

  4. [verwijderd] 16 november 2003 21:37
    At what age do attacks of Hereditary Angioedema start?

    The age of HAE onset varies considerably, however, in one study, one half of the patients reported onset of their symptoms by the age of seven, and over two thirds became symptomatic by the age of thirteen. There also seems to be an increased frequency of attacks during puberty or adolescence.

    How long do Hereditary Angioedema attacks last?

    Patients often report tingling or tightness at the site where edema will occur thirty minutes to several hours later. In some cases, this sensation can be present twelve to twenty four hours before the onset of swelling. Approximately one fourth of HAE patients experience a red blotchy rash both before and during an attack. The edema itself usually lasts for twenty four to seventy two hours, but the length of an attack can range from four hours to four days.

  5. [verwijderd] 16 november 2003 21:38
    When are Hereditary Angioedema attacks considered serious?

    Swelling of the extremities is uncomfortable and, according to some patients, can be painful depending on the location of the edema. Attacks that involve the face and throat must be taken seriously and medical treatment should be sought without delay. Swelling of the throat can close the air passage and cause death by suffocation. The symptoms of an impending airway obstruction include difficulty swallowing and a change in voice pitch.

    Abdominal attacks cause severe pain, nausea, vomiting, and watery diarrhea. Some patients require hospitalization for low blood pressure, dehydration, and pain management. As noted above, abdominal attacks can mimic a surgical abdomen and many patients have been subjected to unnecessary exploratory surgery.

    How is Hereditary Angioedema treated?

    There are three types of therapy for HAE patients.
    1). Long term preventive treatment
    2). Short term preventive treatment
    3). Treatment of acute attacks

    Long term therapy is not required for all HAE patients. In some patients attacks are mild or infrequent and no long-term therapy is required. Clinicians generally recommend long term therapy for patients who experience more than one attack per month, or who believe that the disease significantly interferes with their life style. The present drugs of choice for long-term therapy are 17 alpha alkylated androgens such as stanozolol (winstrol), danazol, and oxandrolone (oxandrin). These agents produce an increase in C1-inhibitor levels, but the exact mechanism of how they do so has not been precisely defined. Some patients report success with a class of drugs called antifibrinolytics, but their use has largely been abandoned because androgens have proven more effective.

    The medical literature and practitioner experience confirms that corticosteriods (prednisone), antihistamines, and epinephrine are not effective in treating angioedema created by C1-inhibitor deficiency. However, a recent study noted success using inhaled epinephrine to prevent complete airway closure.

  6. [verwijderd] 16 november 2003 21:39
    -term therapy is necessary for patients who do not require ongoing treatment but are facing dental procedures or elective surgeries. Current practice calls for daily androgen therapy, during the week prior to surgery. For emergency procedures, fresh frozen plasma (preferably a product that has been treated to inactivate viruses) can be used to prevent attacks.

    There is no currently approved treatment for acute attacks available in the United States. Fresh frozen plasma is used effectively by some clinicians, but this therapy is considered controversial because of a theoretical chance for attack exacerbation. C1-inhibitor concentrate is the treatment of choice for acute attacks of HAE and has been available to patients in Europe for over a decade. C1 inhibitor concentrate treatment resolves the angioedema in thirty minutes to two hours with complete remission in twenty-four hours. C1-inhibitor concentrate has not yet been approved for use in the United States notwithstanding a double blind randomized crossover study published in the New England Journal of Medicine (circa 1996) that concluded it was safe and effective for both prevention and acute attack therapy. A C1-inhibitor concentrate phase III clinical trial sponsored by Baxter Hyland Immuno is now underway in various centers throughout the United States.

    The absence of an effective acute attack therapy limits clinicians to providing supportive care. Maintaining an open airway is the primary concern for patients with laryngeal edema. Because gastrointestinal edema usually involves excruciating pain and frequent vomiting, therapy should include aggressive pain management and fluid replacement. Clinicians report that Zofran, compazine, and phernergan are effective in reducing nausea and vomiting, while either morphine, demerol, dilaudid, darvocet, or stadol can be used to relieve attack related abdominal pain. Unfortunately, the HAE literature in the mid seventies made reference to patients who developed narcotic dependency and this observation was repeated in subsequent papers. Many HAE patients have noted that physicians are often wary of prescribing pain medicine for painful abdominal attacks, and this could be due to reports contained in the literature. Any notion that the HAE population suffers from widespread narcotics addiction has been discredited by the experience of researchers who have treated relatively large numbers of patients.

  7. [verwijderd] 16 november 2003 21:40
    Are there any new treatments on the horizon?

    As noted above, the treatment of choice, C1-inhibitor concentrate, is in final clinical trials and, hopefully, will be approved in the United States sometime in 2001. A partnership between Baxter Hyland Immuno and Pharming NV has been formed and pre clinical studies are underway to obtain recombinant C1-inhibitor from rabbits who have been genetically altered to produce this human compatible protein in their milk. The biotechnology companies Genzyme and Dyax have recently completed a phase I clinical trial testing their propriety kallikrein inhibitor for acute attack management. The Complement Center at the University of Iowa has completed a FDA funded clinical trial testing the efficacy of the anti coagulant heparin for preventing HAE attacks. Italian doctors who treat a large population of HAE patients are hoping they can soon begin to test whether or not a new class of drugs called bradykinin receptor antagonist have a role in treating acute attacks.

    What treatments are available for children who have Hereditary Angioedema?

    Fortunately, most prepubescent children with HAE do not suffer from frequent attacks and infrequent flares affecting the abdomen can be managed by using pain relievers and anti nausea agents. The small number of severely affected children who experience frequent and severe attacks must be managed on a case by case basis. While C1-inhibitor would be the treatment of choice for eradicating acute attacks, it is currently not commercially available in the United States.

    The HAE literature suggests that a class of drugs called antifibrinolytics (Amicar is the only drug in this class available in the US) should be the first drug tried if the parents and physician agree that ongoing treatment is warranted. There are a few reports in the literature of children who did not respond to antifibrinolytics, but have been treated successfully with androgens. A recent case study chronicles the successful use of oxandrolone in treating a severely affected 14-year-old girl. According to the authors of the study, oxandrolone is the only drug in its class that has approved pediatric use. The report noted that oxandrolone reduced the frequency and severity of attacks and no side affects were observed after one year of therapy.

  8. [verwijderd] 16 november 2003 21:41
    Disclaimer: The information, including opinions and recommendations, contained in this document is for educational purposes
    only. Such information is not intended to be a substitute for professional medical advice, diagnosis or treatment. No one
    should act upon any information provided in this document without first seeking medical advice from a qualified medical
    physician. Information derived from the internet or emails, no matter how accurate or relevant are no substitute for competent
    medical care.
  9. [verwijderd] 16 november 2003 22:49
    Inmiddels staat DYAX nu hier:

    Dyax inks partnership with Genzyme over drug
    Boston Business Journal
    A division of Cambridge-based Genzyme Corp. has taken a 50 percent stake in a Phase II drug being developed by Cambridge-based Dyax Corp. for hereditary angioedema, a rare, chronic disease that causes sudden swelling in various parts of the body.


    Genzyme Corp.'s Genzyme General division will pay Dyax milestone fees as the drug, DX-88, enters Phase III trials. If the drug is approved for market, Genzyme will be responsible for all commercialization costs. Dyax obtained orphan drug status for the drug, so the companies will have exclusive market rights in the United States for up to seven years.

    In a statement released Tuesday, Genzyme made the investment based on positive Phase II data of patients taking the drug in Europe. A Phase II trial is underway in the United States. They will share the profits.

11 Posts
|Omhoog ↑

Meedoen aan de discussie?

Word nu gratis lid of log in met je emailadres en wachtwoord.

Direct naar Forum

Premium

Markt lijkt groeipotentieel Pharming te onderschatten

Het laatste advies leest u als abonnee van IEX Premium

Inloggen Word Abonnee

Pharming Nieuws

  1. 10 mei RBC verlaagt koersdoel Pharming
  2. 08 mei Update: Flinke omzetstijging Pharming 2
  3. 08 mei AEX houdt 900 punten waarschijnlijk niet vast, zorgen Ahold, Pharming en SBM voor spektakel? 2
  4. 19 apr Pharming lost voor 123 miljoen euro aan obligaties af
  5. 18 apr Pharming rondt plaatsing obligaties af 5
  6. 18 apr Pharming vervangt converteerbare lening 6
  7. 08 apr Pharming rondt rekrutering voor klinische studie leniolisib met kinderen af
  8. 14 mrt CEO: Pharming haalt omzet uit steeds meer landen 2
  9. 14 mrt Lichte plus voor AEX voorzien; zullen Pharming en Basic-Fit de boel opschudden?
  10. 14 mrt Pharming rekent ook voor 2024 op dubbelcijferige omzetgroei 2

Lees verder op het IEX netwerk Let op: Artikelen linken naar andere sites

Gesponsorde links

Meld u aan voor de dagelijkse Beursupdate

Dagelijks een update van het laatste beursnieuws en beleggingskansen in uw mailbox!