PNH Features

AATD Features, CF Features, Features, hATTR Features, LAL-D Features, MF Features, MS Features, MTC Features, NMOSD Features, PNH Features, Pompe Features, SCD Features, SM Features

Omidubicel Shows Promise as Replacement for Umbilical Cord Blood in Stem Cell Transplants

Larry Luxner

January 25, 2023

Gamida Cell is gearing up to begin sales of omidubicel from Israel to US hospitals for use in stem cell transplants, pending FDA approval of the therapy.

CAD Features, CCA Features, CF Features, Features, HAE Features, hATTR Features, HEM Features, IPF Features, LAL-D Features, LCFAOD Features, PNH Features, Pompe Features, SCD Features

At 40, Orphan Drug Act Enjoys Rare Bipartisan Support

Larry Luxner

January 18, 2023

Rare disease advocates are marking the 40th anniversary of the landmark Orphan Drug Act, which has led to over 1100 approvals for new therapies.

ALGS Features, DLBCL Features, DMD Features, Features, hATTR Features, HEM Features, MG Features, NMOSD Features, PAH Features, PNH Features, Pompe Features, SM Features

Ambitious NCATS Bespoke Gene Therapy Consortium Targets Ultrarare Diseases

Larry Luxner

December 29, 2022

The Bespoke Gene Therapy Consortium (BGTC) is an ambitious public-private partnership that aims to develop gene therapies for ultrarare diseases.

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PNH Features

Omidubicel Shows Promise as Replacement for Umbilical Cord Blood in Stem Cell Transplants

At 40, Orphan Drug Act Enjoys Rare Bipartisan Support

Ambitious NCATS Bespoke Gene Therapy Consortium Targets Ultrarare Diseases

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International Sumaira Foundation Aims to Stop Frequent Misdiagnosis of NMOSD