Press Releases on April 11, 2024 - EIN Presswire - Press Release Distribution Service

Published on April 11, 2024 - 16:33 GMT

Orphan designation: cutamesine alpha-thalassaemia X-linked intellectual disability syndrome (due to mutations in the ATRX gene), ...

This medicine was designated as an orphan medicine for the treatment of alpha-thalassaemia X-linked intellectual disability syndrome (due to mutations in the ATRX gene) on 12 January 2024. This means that the developer will receive scientific and …

Distribution channels: Healthcare & Pharmaceuticals Industry

Published on April 11, 2024 - 16:33 GMT

Orphan designation: Adeno-associated viral vector serotype 9 containing the human MECP2 gene, an intron encoding a miRNA ...

This medicine was designated as an orphan medicine for treatment of the Rett syndrome on 12 January 2024. This means that the developer will receive scientific and regulatory support from EMA to advance their medicine to the stage where they can apply for …

Distribution channels: Healthcare & Pharmaceuticals Industry

Published on April 11, 2024 - 16:33 GMT

Orphan designation: Ziftomenib Treatment of acute myeloid leukaemia, 12/01/2024 Positive

This medicine was designated as an orphan medicine for the treatment of acute myeloid leukaemia on 12 January 2024. This means that the developer will receive scientific and regulatory support from EMA to advance their medicine to the stage where they can …

Distribution channels: Healthcare & Pharmaceuticals Industry

Published on April 11, 2024 - 16:33 GMT

Orphan designation: Recombinant human heparan-N-sulfatase Treatment of mucopolysaccharidosis type IIIA (Sanfilippo A syndrome), 12/...

This medicine was designated as an orphan medicine for the treatment of Mucopolysaccharidosis type IIIA (Sanfilippo A syndrome) on 12 January 2024. This means that the developer will receive scientific and regulatory support from EMA to advance their …

Distribution channels: Healthcare & Pharmaceuticals Industry

Published on April 11, 2024 - 16:33 GMT

Orphan designation: Gorilla adenovirus vector expressing HPV6 and HPV11 antigens Treatment of recurrent respiratory papillomatosis,...

This medicine was designated as an orphan medicine for the treatment of recurrent respiratory papillomatosis on 12 January 2024. This means that the developer will receive scientific and regulatory support from EMA to advance their medicine to the stage …

Distribution channels: Healthcare & Pharmaceuticals Industry

Published on April 11, 2024 - 16:33 GMT

Orphan designation: Human IgG1 monoclonal antibody targeting amyloid transthyretin Treatment of ATTR amyloidosis, 19/02/2024 ...

This medicine was designated as an orphan medicine for the treatment of ATTR amyloidosis on 19 February 2024. This means that the developer will receive scientific and regulatory support from EMA to advance their medicine to the stage where they can apply …

Distribution channels: Healthcare & Pharmaceuticals Industry

Published on April 11, 2024 - 16:33 GMT

Orphan designation: Donidalorsen Treatment of hereditary angioedema, 19/02/2024 Positive

This medicine was designated as an orphan medicine for the treatment of hereditary angioedema on 19 February 2024. This means that the developer will receive scientific and regulatory support from EMA to advance their medicine to the stage where they can …

Distribution channels: Healthcare & Pharmaceuticals Industry

Published on April 11, 2024 - 16:33 GMT

Orphan designation: Autologous CD3-positive T-cells expressing a chimeric antigen receptor against B cell maturation agent ...

This medicine was designated as an orphan medicine for the treatment of AL amyloidosis on 19 February 2024. This means that the developer will receive scientific and regulatory support from EMA to advance their medicine to the stage where they can apply …

Distribution channels: Healthcare & Pharmaceuticals Industry