Familial Dysautonomia (FD)

The Israeli Center for Familial Dysautonomia (FD)

The Israeli Center for Familial Dysautonomia (FD) is located at the Mt. Scopus Campus of Hadassah Medical Center. The center provides counseling, treatment, daycare and monitoring services to patients from Israel and abroad who suffer from FD. The center also provides information, social services and nutrition counseling for patients.

The Center for Familial Dysautonomia was established in Hadassah’s Department of Pediatrics in 1981. Since its establishment it has treated over 200 patients. Patients come from all over the world to be treated at the center, including Russia, Turkey, Australia, Germany, England and the US.

The center provides clinical monitoring, referrals for hospitalizatiot, treatment for patients in the hospital, round-the-clock counseling, doctor visits and counseling for communities. The center is in contact with attending physicians around the country, patients, and para-medical staff.

The information below was provided by the center:

What is Familial Dysautonomia?
Familial Dysautonomia (FD) is a severe chronic condition which affects most of the body’s systems. FD requires constant monitoring and medical care, as well as cooperation between different medical teams and para-medical staff.

A diagnosis is made based on clinical and laboratory tests, which include a genetic test. Our center works together with the Center for Dysautonomia at New York University, headed by Dr. P. Axelrod. The cooperation between the two centers includes both clinical cooperation and research.

Diagnosis of Familial Dysautonomia
A doctor will diagnose a patient following an anamnesis and tests which point to problems in different body systems.

Anamnesis
Anamesis is the patient Jewish of Ashkenazic descent? Did the patient weigh 400 gram less than the average weight at birth?

Physical Examination
There is a typical ‘look’ which indicates FD – asymmetry of the face, tense facial expression, thin lips, strabismus, hypotonia, drooling.

Histamine Examination
For a healthy person, a sub-dermal injection of histamine will produce pain and raise a red flare in the skin. However, if histamine is injected into the skin of someone with FD, there will be no redness surrounding the flare.

Methacholine Testing
A low concentration of methacholine is injected into the conjunctiva sac. This injection will cause an FD patient’s pupil to narrow. This is due to an overreaction of the parasympathetic nervous system, which is damaged.

Denervation Hypersensitivity
The same reaction can be caused by sprinkling pillocarpine into the conjunctiva sac.

Genetic Testing
FD is a recessive autosomal disease. The gene responsible for FD was discovered to be on chromosome 9. Today, it is possible to determine who is a carrier for the disease (Ashkenazic Jews have a 1 in 27 chance of being carriers). Additionally, prenatal genetic testing can be done. These genetic tests are available in most medical centers in Israel. Couples where one or both partners are of Ashkenazic descent must be tested for the gene. We also recommend testing for couples who are not Ashkenazi (there have been cases of carriers among Sephardic Jews too).

If both parents are carriers, they have a 75% chance of having a healthy baby. Early prenatal genetic testing can help the parents reach any decisions they need to regarding the pregnancy. Nowadays, ivf treatments allow us to ensure a healthy baby for carrier parents.

Genetics of Familial Dysautonomia
FD, also called the Riley-Day Syndrome was first reported in 1949 by two physicians, Drs. Riley and Day. The disease mainly affects the autonomous and sensory nervous systems. The autonomic nervous system is responsible for carrying out involuntary functions of the body, such as regulating blood pressure, swallowing, sweating, tearing and more. A defect in the system influences the functionality of most of the body.

The discovery of the location of the FD mutation on chromosome 9 was the result of cooperation between clinical centers in Israel and the US in addition to Dr. James Gasella of Massachusetts General Hospital in Boston. In 2001, the exact location of the gene was found by two different research groups, one led by Dr. Susan Slaugenhaupt of Massachusetts General Hospital and Dr. Berish Rubin of Fordham University in New York. Within a few short months, the number of FD-affected patients being born had decreased. Following the discovery of the gene, research is now focused on finding possible cures for the mutation.

Incidence of FD
The disease is only relatively common among Ashkenazi Jews. In a 1987 research, the incidence was found to be 1:3,700. Following a large-scale research, the carrier rate among Polish Jews is higher, 1:18, as opposed to non-Polish Ashkenazi Jews, which is 1:99. Ashkenazi Jews with partial Polish heritage have a 1:56 incidence.

A third of all patients live in the New York area, a third live in Israel and the rest were found in different areas around the US, Canada, South America, South Africa and Europe. Nowadays, there are around 400 patients (122 in Israel, 170 in the US, 15 in EU and the rest in other countries).

Symptoms of FD

Digestive System

  • Low sucking ability
  • Lack of coordination swallowing
  • Aspiration in the lung, especially when eating fluids.
  • G-E Reflux
  • Vomiting\
  • Dysautonomia crises – vomiting attakcs, high blood pressure, excessive sweating, redness on cheeks. These crises can last a few minutes, hours, days or even weeks. May require hospitalization.

Pulmonary System

  • Aspiration pneumonias (lung infections caused by the misdirection of fluid or food when swallowing)
  • Inability to breath while crying (can cause fainting)
  • Restrictive pulmonary disease
  • Central Sleep Apnea

Vision

  • Lack of tears after 3 months of age (up until the age of 3 months this is normal)
  • Dry eyes
  • Strabismus
  • Gradual vision loss

Cardiovascular System

  • Blood pressure changes, causing dizziness
  • Heart rhythm problems

Musculoskeletal System

  • Curved spine
  • Osteoporosis
  • Orthopedic problems

Nervous System

  • Hypotonia during childhood
  • Lack of coordination
  • Speech problems
  • Differences in taste
  • Inability to feel pain
  • Delayed milestones

Urinary System

  • Inability to control urine
  • Decrease in kidney function (among adults)
  • Late sexual maturation

Skin

  • High frequency of seborrheic dermatitis
  • Extremities are often cold (lack of proper blood flow)
    Blotching

Psycho-Social

  • Emotionally instable

Different patients suffer from different symptoms at different times during their lives.

The Israel Center for Familial Dysautonomia is located on the Mt. Scopus campus of Hadassah Medical Center. It is located on the ground floor. The center is headed by Prof. Channa Maayan.

The center is open Sunday – Tuesday, from 9:00 am.
To make an appointment, please call 02-5844510 or 050-7874428 (Prof. Maayan).