Pompe disease

We’re all in this for good.

By sharing your stories and data, you will:
  • help each other live better and uncover the best ways to manage your health today
  • help researchers shorten the path to new treatments tomorrow

How much good can your data do? A whole lot, as co-founder Jamie Heywood explains in this video.

What is Pompe disease?

Pompe disease, also known as glycogen storage disease type II, is a condition characterized predominantly by general muscle weakness. It may occur in infants or children and adults. In the infant form, the heart is significantly affect; in the adult form, the limbs and diaphragm are affected.

Common symptoms reported by people with Pompe disease

Common symptoms
How bad it is
What people are taking for it

Reports may be affected by other conditions and/or medication side effects. We ask about general symptoms (anxious mood, depressed mood, fatigue, pain, and stress) regardless of condition.
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Treatments taken by people with Pompe disease

Treatment name(s)
Type
How many have tried
Tried for
Treatment name(s)

(Lumizyme)
Type
Prescription Drug
How many have tried
2
Tried for
Pompe disease (2)

Data from patients with Pompe disease, who reported starting treatments within the last 5 years.
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Compare treatments taken by people with Pompe disease

Treatment name(s)
Perceived effectiveness
Side effects
Total evaluations
Tried for
Treatment name(s)

(Lumizyme)
Perceived effectiveness
for Pompe disease (2 evaluations)
Side effects
headaches itching
Total evaluations
2
Tried for
Pompe disease (2)
  • Major
  • Moderate
  • Slight
  • None
  • Can't tell
  • Severe
  • Moderate
  • Mild
  • None

Data from patients with Pompe disease, who reported starting treatments within the last 5 years.
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Who has Pompe disease on PatientsLikeMe?

  • 0 new patients joined this month
  • 63 say Pompe disease is their primary condition

Age
Age Proportion # of patients
<20 5
20s 1
30s 12
40s 13
50s 22
60s 5
70+ 5
Age at first symptom
Age at first symptom Proportion # of patients
0-19 years 7
20-29 years 3
30-39 years 7
40-49 years 2
50-59 years 2
60-69 years 1
70+ years 0

Distribution of sex

Sex
Sex Proportion # of patients
Male 16
Female 46

Types
Condition Types Proportion # of patients
Adult onset 45
Infantile onset 2
Juvenile onset 3
Diagnosis status
Diagnosis status Proportion # of patients
Diagnosed 25
Not Diagnosed 1

These charts show data from Pompe disease patients who have completed their condition history
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