I kēia lā, mahalo ka TSC Alliance® i ka ʻae ʻana o ka US Food and Drug Administration (FDA) iā HYFTOR™, ʻo ia ka lāʻau lapaʻau topical mua i ʻae ʻia e FDA no ka angiofibromas maka i nā pākeke a me nā keiki ʻeono makahiki a ʻoi paha me ka tuberous sclerosis complex (TSC) . ʻO HYFTOR™, i hana ʻia e Nobelpharma America, LLC, he Orphan Drug Status no kēia hōʻailona.
"Hoʻokipa maoli ka TSC Alliance i kēia koho lapaʻau kumuhana no nā angiofibromas," wahi a Kari Luther Rosbeck, Pelekikena & Luna Nui o ka TSC Alliance. "No ka mea e hoʻopilikia pinepine lākou i ka helehelena o kekahi a hiki ke hoʻokahe koko, hiki i kēia lapaʻau ke hōʻemi maoli i ka hopena o kēia hōʻike i nā pākeke a me nā keiki me TSC. Mahalo mākou no ka hoʻokō ʻana o Nobelpharma i ke kaiāulu TSC.
ʻO ka TSC kahi maʻi maʻi maʻi ʻaʻole e hoʻokumu ʻia i loko o nā kino koʻikoʻi, me ka ʻili. ʻO nā Angiofibromas i hoʻokumu ʻia e ka TSC he mau puʻupuʻu liʻiliʻi i hoʻopuehu pinepine ʻia ma ka ʻaoʻao waena, ʻoi aku ka nui ma ka ihu a me nā papalina, a hui pinepine ʻia i loko o nā awāwa ma ka ʻaoʻao o ka ihu. ʻOi aku ka liʻiliʻi o nā Angiofibromas ma mua o ka peppercorn, akā hiki ke ulu nui. He ʻili-ʻili paha, ʻulaʻula a ʻulaʻula paha. Loaʻa nā Angiofibromas i ka hapa nui o nā kānaka me ka TSC ma mua o 5 mau makahiki a hiki ke kahe koko. Hiki iā lākou ke loaʻa nā hopena maikaʻi ʻole i ka helehelena a me ke kiʻi ponoʻī, e koi ana i kekahi poʻe me TSC e pale i nā kūlana pilikanaka.
"I ka hui o ka TSC Alliance's 2017 Externally-Led Patient-Focused Drug Development hālāwai, ua lohe pololei mākou mai nā poʻe me TSC i ka pilikia o ke koko o ka maka i hoʻopilikia i ko lākou hiki ke komo i nā haʻuki ikaika," wahi a Steven L. Roberds, PhD, Luna Nui ʻepekema o ka TSC Alliance, "Manaʻolana mākou e kōkua kēia huahana i nā poʻe hou aʻe e ola i ke ola olakino a hauʻoli."
He aha e lawe ʻia mai kēia ʻatikala:
- Angiofibromas caused by TSC are small bumps usually scattered on the central face, especially on the nose and cheeks, and are often clustered in the grooves at the side of the nose.
- “Since they often affect someone’s appearance and can cause bleeding, this treatment has the potential to truly reduce the impact of this manifestation on adults and children with TSC.
- Food and Drug Administration’s (FDA’s) approval of HYFTOR™, which is the first FDA-approved topical treatment for facial angiofibromas in adults and children six years of age or older who have tuberous sclerosis complex (TSC).
