LGMD Muscle Patients Bring Their Drug Demands Back to the Agenda in Turkey

LGMD Muscle Disorders: The Fight Against Rare Diseases

LGMD (Limber Girdle Muscular Dystrophy) muscle diseases are a disease that affects many individuals today. rare diseases It is among. This disease is a genetic disorder that causes muscle weakness and loss over time. There are many patients with this disease in Turkey and the demands of these patients attract the attention of the health system.

What is LGMD and What are its symptoms?

LGMD is a group of genetic diseases characterized by muscle weakness. Symptoms of the disease usually begin to appear between the ages of 8 and 10. These symptoms include muscle weakness, difficulty walking, difficulty climbing stairs ve muscle cramps As the disease progresses, daily living activities become difficult and the patient's quality of life decreases.

Subgroups of LGMD

LGMD is divided into approximately 30 different subgroups. These subgroups differ according to the genetic structure of the disease. For example, different subgroups such as LGMD1 and LGMD2 affect the course of the disease and treatment methods. Each subgroup is based on different gene mutations and therefore the treatment processes also vary.

The Situation of LGMD Patients in Turkey

LGMD patients in Turkey often face significant difficulties due to the inadequacies of the healthcare system. The necessary Gene therapies ve medicines, is often inaccessible to patients. The LGMD Muscle Disease Platform is trying to create significant awareness on this issue and voice the demands of patients.

Patients' Requests and Actions

LGMD patients organize various actions to convey the difficulties and needs they experience during their treatment process to the authorities. For example, in the action organized together with the DMD Association on September 7, patients announced their demands and meetings were held with the Ministry of Health. However, the results were generally not satisfactory.

  • Access to Medicine: One of the biggest demands of patients is to make necessary medications accessible through SSI.
  • Gene Therapies: Approval processes need to be accelerated so that gene therapies performed abroad can also be applied in Turkey.
  • Creating Awareness: Raising awareness about LGMD disease in society will help understand the difficulties experienced by patients.

Gene Therapies and New Hopes

Among the most promising treatment methods for LGMD patients Gene therapies Studies conducted abroad in this area have the potential to change the course of the disease. However, it is of great importance that the necessary approval processes be accelerated for these treatments to be applied in Turkey. Patients are calling on the authorities to make these treatments available in Turkey.

Community Support and Awareness

Understanding the difficulties experienced by LGMD patients and creating social support for this issue is of critical importance. Awareness campaigns make the difficulties experienced by patients visible and contribute to society becoming more sensitive to this issue. Patients, along with their families and friends, expect support from all segments of society.

As a result

LGMD muscle diseases are a significant health problem affecting many individuals in Turkey. Patients' demands for treatment and the inadequacies of the health system in this regard should be addressed as a social responsibility. Gene therapies and accessibility of drugs are of great importance to improve the quality of life of patients. The sensitivity of every segment of society on this issue will help patients' voices to be stronger.