USANew treatments and approvals
Revakinagene taroretcel, an implanted cell therapy for MacTel type 2, approved
The FDA has approved revakinagene taroretcel for macular telangiectasia type 2.
Official drug approvalMacTel
Rare Diseases
News
New treatments, research and health policy from around the world and from Türkiye. Each with its source and level of evidence.
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EUNew treatments and approvals
Once-monthly eplontersen injection approved in the EU for nerve damage in hereditary TTR amyloidosis
The European Commission approved eplontersen for hereditary TTR amyloidosis.
Official drug approvalATTRv amyloidosis
EUNew treatments and approvals
Conditional EU approval for belzutifan for tumors linked to von Hippel-Lindau disease
The EU granted conditional approval to belzutifan, which is taken by mouth.
Official drug approvalVHL
USANew treatments and approvals
Mirdametinib approved in the USA for NF1-related plexiform neurofibromas
The US drug agency (FDA) has approved oral mirdametinib for patients with NF1.
Official drug approvalNF1
USANew treatments and approvals
Crinecerfont, used together with steroids, approved in the USA for classic CAH
On December 13, 2024, the US Food and Drug Administration (FDA) approved a drug called crinecerfont for classic CAH (congenital adrenal hyperplasia).
Official drug approvalCAH