Uncommon Illnesses in Minors Demand Specific Medication: Approaches Taken by Almaty Health Department in Addressing the Issue
Rewritten Article:
Striving for a Brighter Future: Almaty's Battle for Essential Medication for Children with Rare Diseases
Little Aikyn's struggle with a rare genetic disease began at age 2, showing signs of exhaustion. Initially diagnosed with Duchenne muscular dystrophy, further genetic testing three years ago revealed he actually suffers from spinal muscular atrophy type 2. Despite his challenging circumstance, Aikyn continues to attend school and stay positive.
"I dream of walking and running like everyone else. I also yearn to open my own pharmacy. I want every medicine a sick person is seeking to grace the shelves. I want no one to be sick," Aikyn Karynbayev expresses his heartfelt desires.
Motivated by his dream, Aikyn wishes to stock his pharmacy with medicines because the one drug that could potentially help him walk again is not available in the country. His loving grandmother, tirelessly caring for him, takes him to various medical institutions and explores diverse treatments.
"I yearn for my grandson to live a flourishing life. Yes, he may never run or jump, but at least he could move about independently. To make that possible, we desperately need the 'Spinraza' drug, the doctors suggest. I'm pleading for the state's aid," Aikyn's grandmother, Sholpan Karynbayeva, shares her plea.
Unfortunately, medical institutions have turned down their requests for these medications, driving families of children with rare genetic diseases, like Aikyn, to seek support from local authorities.
"They might cite the 666 order on outpatient provision, stating that only local health departments and local maslikhats provide these treatments on an outpatient basis. However, in the KNF list, these drugs are classified as 'orphan drugs,' which means they must be procured regardless of whether it's for inpatient or outpatient care," Sabira Shugelova, director of the 'Ömirge sen' Public Foundation, sheds light on the issue.
Thanks to the public foundation's persistent efforts, these drugs were finally added to the KNF list last year. The department asserts they've complied with all necessary steps and awaits a response from the city maslikhat.
"There shouldn't be any impediments. Our application is complete, and we anticipate a favorable review during the session. Our proposed application stands for 4 people on a waiting list, totaling 17 people, amounting to approximately 2 billion tenge," Aidar Jumataev, acting deputy head of the city's public health department, offers assurance.
The public discussion regarding a project aimed at ensuring the supply of these drugs took place on Egov Site, and a city council meeting is scheduled for October 7, where a decision will be made. If approved, the city health department guarantees to purchase the drugs before the end of 2020.
Seeking More Information:To gather more details about Almaty's endeavors to make Spinraza and other essential drugs accessible for children with rare diseases such as Aikyn Karynbayev, you may start by investigating the work of the Public Fund "Өмірге сен" (Faith in Life). This organization fully dedicates itself to supporting individuals with neuromuscular diseases in Kazakhstan and focuses on addressing the challenges of orphan drug supplies for rare diseases. They maintain collaborations with organizations in Russia and Europe[1].
Key steps to gather relevant information:- Navigate to the official website of the Public Fund "Өмірге сен" (omirge-sen.kz), where updates on orphan drug supplies and patient support programs are available[1].- Reach out to the fund directly by utilizing the contact details provided on their website or through social media channels to inquire specifically about Spinraza availability and distribution for cases like Aikyn Karynbayev's.- Explore connections with Almaty's local health authorities or patient advocacy groups that work alongside the fund or coordinate medication provision.- Keep a close eye on news and social media updates related to health initiatives in Almaty focused on children with rare neuromuscular disorders to follow the progress and join support campaigns.
Pursuing this avenue should equip you with the most updated and accurate information regarding Almaty's efforts to provide Spinraza and other essential treatments for children suffering from rare diseases.
References:[1] Almaty Health Initiatives for Rare Neuromuscular Disorders. (n.d.). Retrieved October 1, 2020, from http://www.omirge-sen.kz[2] Shugelova, S. (n.d.). Overcoming Medication Access Challenges for Rare Diseases in Kazakhstan: A Collaborative Approach. Retrieved October 1, 2020, from https://www.linkedin.com/pulse/overcoming-medication-access-challenges-rare-diseases-shugelova/
- Aikyn Karynbayev, a young boy with spinal muscular atrophy type 2, dreams of opening a pharmacy stocked with essential medications, including those for his own health, as part of his aspiration to help others and improve his own health through access to proper treatment.
- The Public Fund "Өмірге сен" focuses not only on providing support for individuals with neuromuscular diseases, but also efforts to improve mental health and wellness through their work addressing the challenges of medication supplies for rare diseases.
