Rare diseases, Rare Disease FAQ - Page 3

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Understanding Vasculitis: Insights on Rare Disease Management and Resources

Vasculitis Hello, Doctor. I would like to inquire about the condition of vasculitis. My sister-in-law is 34 years old and was hospitalized last month due to pneumonia. Later tests revealed that she also has glomerulonephritis. Based on her blood tests, her ANCA levels are elevate...


Dr. Lin Qingyuan Reply:
Since you are already 34 years old, I recommend seeing an adult nephrologist. May I ask where your sister-in-law lives for a proper referral?

[Read More] Understanding Vasculitis: Insights on Rare Disease Management and Resources


Understanding Neonatal Hypoxia: Essential Tests and Considerations

Pediatric Hypoxia Hello Dr. Lin, I apologize for reaching out, but your name is only found among the specialists in rare diseases. My sister gave birth to a baby boy today, and the attending physician mentioned that the baby exhibited signs of respiratory distress after birth, s...


Dr. Lin Guanglin Reply:
Based on your description, it is not possible to determine if there is hypoxia. Currently, regular developmental follow-ups with a pediatrician are sufficient. If there are concerns, a cranial ultrasound can be performed.

[Read More] Understanding Neonatal Hypoxia: Essential Tests and Considerations


Understanding Rare Diseases: Is My Daughter's Condition Tourette Syndrome?

Heart My daughter is 12 years old. Last summer, her right leg started to shake involuntarily. The duration varies; sometimes it stops after 5 minutes, and other times it can last for 25 hours. All physical examinations have come back normal. Could this be Tourette syndrome?


Dr. Chen Shunsheng Reply:
The following conditions can cause tremors in the hands and feet: 1. Tourette syndrome 2. Focal motor epilepsy 3. Peripheral nerve dysfunction: Often seen in various early peripheral neuropathies, commonly accompanied by muscle cramps or twitching symptoms 4. Essential tremor: A ...

[Read More] Understanding Rare Diseases: Is My Daughter's Condition Tourette Syndrome?


Understanding Creutzfeldt-Jakob Disease: Current Treatments and Research Updates

Kujak's syndrome Dr. Chen: Hello, I heard that you are an expert in researching Creutzfeldt-Jakob disease (CJD). My father has been ill since the end of February this year, and it has been five months. He was previously treated at your hospital and is currently in a nursing ...


Dr. Chen Shunsheng Reply:
A potential new treatment for Creutzfeldt-Jakob Disease (CJD) has finally been discovered. CJD and human transmissible spongiform encephalopathies are both fatal conditions and currently classified as untreatable rare diseases. A research group led by Nobel laureate Prusiner at t...

[Read More] Understanding Creutzfeldt-Jakob Disease: Current Treatments and Research Updates


Understanding Crohn's Disease: Why It's Not Classified as a Rare Disease in Taiwan

Crohn's disease I would like to ask Dr. Jian why Crohn's disease is not classified as a rare disease. I have heard from my attending physician that its incidence is very low among East Asians, and it is somewhat related to genetic factors. Is it true that in Taiwan, the...


Dr. Jian Yingxiu Reply:
Currently, the rare diseases defined by the Rare Disease Act refer to diseases with a prevalence rate below the standards set by the central competent authority and recognized by the committee as specified in Article 4, or those designated by the central competent authority due t...

[Read More] Understanding Crohn's Disease: Why It's Not Classified as a Rare Disease in Taiwan


Understanding Rare Diseases: A Journey Through Diagnosis and Symptoms

The difficult next step? Hello Doctor: My illness seems to have started in the first year of junior high school. I don't know why my legs began to feel weak, making it difficult to run or perform any vigorous repetitive movements, and my muscles are prone to strains. Shortly...


Dr. Chen Shunsheng Reply:
I have not seen a patient, so I cannot directly tell you what disease it is. However, I can introduce you to spinal anterior horn cell degeneration disorders: Spinal muscular atrophy (SMA) is a group of diseases caused by the degeneration of anterior horn cells (motor neurons) in...

[Read More] Understanding Rare Diseases: A Journey Through Diagnosis and Symptoms


Understanding Rare Diseases: Why Do I Feel Cold in Winter?

Winter can be cold? Doctor, may I ask... during winter, sometimes I feel extremely cold, to the point of being unable to tolerate it. What could be the underlying condition?


Dr. Jian Yingxiu Reply:
Please visit the Family Medicine outpatient clinic for consultation.

[Read More] Understanding Rare Diseases: Why Do I Feel Cold in Winter?


Improving Quality of Life for Patients with Ataxia: Tips and Treatments

How to improve the condition? Dear Dr. Chen, I would like to express my gratitude to Dr. Lu Ching-Song and his colleagues at Linkou Chang Gung Memorial Hospital for their assistance. My medical record number is 1457244, and it has been confirmed that I am a patient with Spinocer...


Dr. Chen Shunsheng Reply:
There are no specific curative medications available. Typically, antioxidants such as vitamin E and coenzyme Q10 or its derivative idebonone are used for symptomatic treatment. Symptomatic management may involve trialing medications like sodium valproate, baclofen, and L-dopa. Or...

[Read More] Improving Quality of Life for Patients with Ataxia: Tips and Treatments


The Impact of Rare Diseases on Society: Prevalence and Challenges

Rare diseases What impact do rare diseases have on society? What is the average incidence rate per year?


Dr. Jian Yingxiu Reply:
Rare diseases are defined as conditions with extremely low prevalence and a very small number of affected individuals. Most of these are genetic disorders, while some are non-genetic or of unknown origin. According to the U.S. Orphan Drug Act, a disease is considered rare if it a...

[Read More] The Impact of Rare Diseases on Society: Prevalence and Challenges


Can You Get a Health Checkup at Night? Understanding Rare Diseases

Is there a physical examination in the evening? Is there a physical examination in the evening?


Dr. Lai Xiangrong Reply:
There are several types of health examinations: 1. For examination and certification purposes, typically conducted in family medicine, which can also be provided during evening outpatient services. 2. Health check-ups may require scheduling, and the availability varies by hospita...

[Read More] Can You Get a Health Checkup at Night? Understanding Rare Diseases


Understanding Cord Blood Applications for Rare Diseases: Compatibility and Testing

Applications of Umbilical Cord Blood Hello, I would like to inquire whether umbilical cord blood can only be used by individuals who are blood relatives, or what testing methods can determine compatibility. I look forward to your response. Thank you!


Dr. Jian Yingxiu Reply:
Umbilical cord blood transplantation still requires matching, meaning that the donor and recipient must have compatible human leukocyte antigens (HLAs) to avoid rejection. The likelihood of matching is higher among relatives than between non-relatives, resulting in better outcome...

[Read More] Understanding Cord Blood Applications for Rare Diseases: Compatibility and Testing


How to Access Medications for Rare Diseases: A Guide

Rare diseases Hello Dr. Jian, I would like to ask how medications for rare diseases are generally obtained.


Dr. Jian Yingxiu Reply:
At National Taiwan University Hospital, we have some commonly used medications for rare diseases available. If the indications for use are met, prescriptions can be directly filled at our pharmacy by the physician. For non-routine medications, if there is an urgent need, inquirie...

[Read More] How to Access Medications for Rare Diseases: A Guide


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