InternationalResearch and clinical trials
Cemdisiran, given every three months, reduced myasthenia gravis symptoms in a phase 3 trial
The NIMBLE study, published in The Lancet, examined cemdisiran in myasthenia gravis.
Neuromuscular diseases
A disease in which the immune system disrupts the signals between nerves and muscles. The muscles tire quickly and become weak. It often begins with a drooping eyelid and double vision.
Not part of the panel of Türkiye's National Newborn Screening Programme (heel-prick test). Source
Orphanet has a guideline for emergency doctors for this disease (language: German, English, Spanish, French, Italian, Polish). See on Orphanet
This page is translated from the Turkish original.
This disease is followed through the “Neuromuscular diseases” group.
Myasthenia gravis is a disease in which the immune system disrupts the signals between nerves and muscles. Because of this, the muscles tire quickly and become weak. The weakness increases with activity and decreases with rest. It is a long-lasting disease; there may be flare-ups and quiet periods.
Symptoms often begin with the eye muscles. They can change during the day and from day to day.
Blood tests and tests that measure nerve-to-muscle signals are used for the diagnosis. A CT scan or MRI may be done to look at the thymus gland in the chest. The thymus is a gland involved in the immune system.
Medicines that reduce muscle weakness are used. Some people may need surgery to remove the thymus gland. Reducing tiredness and stress can ease the symptoms. Severe cases can be serious; but most people have a normal life expectancy. The doctor who follows the disease decides which treatment is suitable.
Myasthenia gravis is often not inherited. Most people with the disease have no one else in the family with it. In a small number of families, more than one person can be affected; it is helpful to talk about this with the doctor.
Source: MedlinePlus Genetics – Myasthenia gravis · NHS – Myasthenia gravis
These facts are taken from Orphanet and MedlinePlus records (last checked: October 7, 2026). Frequency values are approximate ranges.
Non-profit patient and family organizations in Türkiye working on this disease or disease group. Links are for information; Doktorclub is not affiliated with these organizations and does not endorse them.
For all rare diseases: Nadir Hastalıklar Federasyonu
Links checked on October 8, 2026.
Doktorclub does not diagnose, recommend treatments or refer you to hospitals or doctors. These pages are meant to help you talk with your doctor.
InternationalResearch and clinical trials
The NIMBLE study, published in The Lancet, examined cemdisiran in myasthenia gravis.
EUNew treatments and approvals
On November 28, 2025, the European Commission approved nipocalimab for generalized myasthenia gravis.
A clinical trial is a regulated study that tests whether a new treatment is safe and whether it works, in patients who volunteer. Most trials look for a certain age, genetic change and health condition. Taking part is voluntary.
Questions you can ask your doctor
ClinicalTrials.gov lists 100 active interventional studies for this disease. 57 are recruiting now; 7 have sites in Türkiye. Last checked: October 7, 2026.
Selected records (studies with a site in Türkiye first):
Late stage: the effect is being compared in a large group · Phase 3 · investigational (experimental)RecruitingSite in TürkiyeNCT06463587
Middle stage: the effect is being tested in a small group · Phase 2 · investigational (experimental)RecruitingSite in TürkiyeNCT07501702
Late stage: the effect is being compared in a large group · Phase 2 / Phase 3 · investigational (experimental)Active, not recruitingSite in TürkiyeNCT06149559
Late stage: the effect is being compared in a large group · Phase 3 · investigational (experimental)Enrolling by invitationSite in TürkiyeNCT06540144
Late stage: the effect is being compared in a large group · Phase 3 · investigational (experimental)Active, not recruitingSite in TürkiyeNCT05070858
See all studies on ClinicalTrials.gov · Joining clinical trials
InternationalResearch and clinical trials
The HIMALAYA study, published in The Lancet Neurology, examined an experimental drug in patients with ALS (amyotrophic lateral sclerosis).
TürkiyeResearch and clinical trials
A multicenter study in Istanbul compared patients with Guillain-Barré syndrome.
USANew treatments and approvals
The US Food and Drug Administration (FDA) approved levacetylleucine for balance and walking problems in ataxia-telangiectasia (A-T).
These codes identify the disease in international classifications and databases. They can help when you talk with your doctor or look for reliable sources.
Orphanet code: ORPHA:589 · ICD-11 8C60
Codes checked against WHO ICD-11 and Orphanet on October 6, 2026.
This page provides general information and does not replace diagnosis or treatment. Talk to your doctor about symptoms, diagnosis and treatment.