Bimwe mu biyiranga harimo gucika intege kw’imikaya no kugabanyuka k’uburemere bwayo, kugira ibibazo byo guhumeka, kunanirwa kugenda, kugorwa no kurya n’ibindi birimo kwangirika k’urutirigongo ku buryo rwiheta.

Ubusanzwe nta miti ihari ivura iyi ndwara usibye kuba nk’uyitahuweho mu bihugu bitaratera imbere agororwa ingingo (Physiotherapy) gusa. Bishyira mu kaga ubuzima bwe nk’uko byagarutsweho n’umushakashatsi akaba n’Umwarimu muri Kaminuza ya Edinburgh yo mu Bwongereza,Tom Gillingwater.

Hari nk’aho avuga ko iyi ndwara izahaza abana ku buryo batanakunze kugira amahirwe yo kugaruka mu buzima n’iyo bahawe serivisi y’ubuvuzi ndenzaminsi ihabwa abasigaje nibura ibyumweru 12 ngo bapfe (palliative care).

N’ubwo bimeze bityo, Urubuga Muscle & Nerve rwagaragaje ko ubuvuzi bwo gusimbuza ingirabuzima fatizo (Gene Therapy) bwifashishishwa mu bihugu byateye imbere, butanga icyireze ku bana bato bafite ubu burwayi.

Nko guhera mu mpera za 2020 kugeza mu ntangiriro za 2022, abana 25 babuherewe mu Bitaro bya Al Jalila Children’s Specialty i Dubai kandi bamererwa neza n’ubwo hari izindi ngaruka zabwo ku wabuhawe.

Ubu buvuzi buzwi nka ‘Zolgensma’ bwavumbuwe na Sosiyete Novartis International AG (Novartis) y’Abasuwisi, imaze kubaka izina mu gukora imiti guhera mu 1996, buhabwa cyane abana bari munsi y’imyaka ibiri.

Uhawe ubuvuzi bwa ‘Zolgensma’ aterwa umuti usukika mu mujyana (Vein) hagakoreshwa ingano ijyanye n’ibilo bye, ku buryo ingano y’umuti ukenewe mu mubiri we idashobora kuba nyinshi cyangwa nke ngo bibe byatanga umusaruro uteri witezwe.

Buhabwa abana bari munsi y’imyaka ibiri bwemejwe n’ Ikigo gishinzwe kugenzura ibiribwa n’imiti muri Leta Zunze Ubumwe za Amerika, FDA mu 2019, kinagaragaza ko ari bumwe mu buhenze ku Isi aho ubuhawe yishyura agera kuri miliyoni 2$, ni ukuvuga asaga miliyari 2 Frw.

Urugero rwa hafi ni nk’aho abaturage ba Leta Zunze Ubumwe z’Abarabu muri Mutarama 2023 bakusanyije miliyoni 1,8$ (ni ukuvuga asaga miliyari 2,1 Frw) kugira ngo umwana w’umwaka n’amezi arindwi wari ufite ikibazo cyo guhumeka bitewe n’indwara ya Spinal muscular atrophy abuhabwe.

Ubusanzwe iyi ndwara iri mu byiciro bine, aho nko mu cya mbere umwana wayivukanye itangira kugaragara mu mezi atandatu ndetse bigatuma atazigera abasha kugenda, ikaba yanamukururira urupfu ku bwo kwibasirwa n’ibibazo byo guhumeka bigoranye. Abana yibasiye muri iki kigero ntibakunze kubaho imyaka irenze ibiri.

Icyiciro cya kabiri ho ishobora kugaragara ku mwana nyuma y’icyo gihe n’ubwo bidakunze kubaho, aho umwana ashobora kubasha kwiyicaza ariko ntabashe kugenda adahawe ubufasha bikaba byanamuviramo kugira ubundi bumuga butandukanye.

Mu cya gatatu cyo abana bafite ubu burwayi bashobora kubasha kugenda neza cyangwa rimwe na rimwe bakagira ibibazo mu migendere bishobora gutuma bagendera mu igare ryifashishwa n’abantu bafite ubumuga, mu gihe icyiciro cya kane cy’iyi ndwara cyo kidakunze kugaragara aho ifata uwamaze gukura ukiri mu myaka y’urubyiruko.

Urubuga rwa American Congress of Obstetricians and Gynecologists mu 2018 rwatangaje ko umwana umwe mu bari hagati ya 6.000 na 10.000 bavuka, agira ubu burwayi bwa Spinal muscular atrophy.

Ni mu gihe iyo ababyeyi bombi bafite ingirangingo fatizo (genes) zishobora guhererekanya iyi ndwara baba bafite ibyago bingana na ¼ byo kubyarana umwana na we akazayirwara.

Muri Werurwe 2023, uruguga rwa Global Genes rwagaragaje ko icyo gihe ubuvuzi bwa Zolgensma bwari bumaze kwemezwa mu bihugu 47, bwarahawe nibura abarwayi bwa Spinal muscular atrophy basaga 3,000 bo ku Isi yose.

Zimwe mu ngaruka zigera ku wahawe ubwo buvuzi harimo kuba yagira ibibazo by’umwijima, akaba ari byiza ko nk’umwana wabuhawe aba agomba kujya akorerwa isuzumwa ryawo kenshi, mu gihe agaragaza ibimenyetso birimo kuba yatangira guhinduka kw’ibara ry’uruhu n’amaso bigasa umuhondo, kuruka bya hato na hato no kujya yibasirwa n’umunaniro udasobanutse.

Ubuvuzi bwo gusimbuza ingirabuzima fatizo (Gene Therapy), kugeza ubu ni bwo butanga icyizere ku bana barwaye kunanuka kw’imikaya y’urutirigongo