Press Releases on April 8, 2024 - EIN Presswire - Press Release Distribution Service

Published on April 8, 2024 - 16:03 GMT

Orphan designation: rovatirelin Treatment of spinocerebellar ataxia, 11/11/2022 Positive

Overview This medicine was designated as an orphan medicine for the treatment of spinocerebellar ataxia in the European Union on 11 November 2022. This means that the developer will receive scientific and regulatory support from EMA to advance their …

Distribution channels: Healthcare & Pharmaceuticals Industry

Published on April 8, 2024 - 16:03 GMT

Orphan designation: Lactobacillus plantarum Treatment of amyotrophic lateral sclerosis, 16/12/2019 Positive

At the time of designation, amyotrophic lateral sclerosis affected approximately 1 in 10,000 people in the European Union (EU). This was equivalent to a total of around 52,000 people*, and is below the ceiling for orphan designation, which is 5 people in …

Distribution channels: Healthcare & Pharmaceuticals Industry

Published on April 8, 2024 - 16:03 GMT

Orphan designation: Adeno-associated viral vector serotype 8 encoding B-domain deleted liver specific codon optimized ...

This medicine, also known as ASC618, is made of a virus that has been modified to contain a gene that encodes for factor VIII, the protein that is lacking in patients with haemophilia A. This protein is necessary for the formation of blood clots. When …

Distribution channels: Healthcare & Pharmaceuticals Industry

Published on April 8, 2024 - 16:03 GMT

Orphan designation: Alisitol, retinol palmitate, zinc gluconate Treatment of microvillus inclusion disease, 13/11/2020 Positive

At the time of designation, microvillus inclusion disease affected less than 0.01 in 10,000 people in the European Union (EU). This was equivalent to a total of fewer than 500 people*, and is below the ceiling for orphan designation, which is 5 people in …

Distribution channels: Healthcare & Pharmaceuticals Industry

Published on April 8, 2024 - 16:03 GMT

Orphan designation: imatinib Treatment of pulmonary arterial hypertension, 21/06/2021 Positive

Overview This medicine was designated as an orphan medicine for the treatment of pulmonary arterial hypertension (PAH) in the European Union on 21 June 2021. This means that the developer will receive scientific and regulatory support from EMA to advance …

Distribution channels: Healthcare & Pharmaceuticals Industry

Published on April 8, 2024 - 16:03 GMT

Orphan designation: idebenone Treatment of Leber's hereditary optic neuropathy, 16/02/2007 Positive

At the time of designation, Leber's hereditary optic neuropathy affected less than 1 in 10,000 people in the European Union (EU)*. This is equivalent to a total of fewer than 46,000 people, and is below the ceiling for orphan designation, which is 5 …

Distribution channels: Healthcare & Pharmaceuticals Industry

Published on April 8, 2024 - 16:03 GMT

Orphan designation: Autologous CD34+ cells transduced with a lentiviral RNA vector that results in integrated cDNA encoding for ...

Cystinosis is caused by a defect in the gene for cystinosin, a protein essential for normal cell function. The medicine, also known as AVR-RD-O4, is a gene therapy, produced from immature blood (stem) cells collected from the patient. A therapeutic gene ( …

Distribution channels: Healthcare & Pharmaceuticals Industry

Published on April 8, 2024 - 16:03 GMT

Orphan designation: Olorofim Treatment of invasive Scopulariopsis, 14/01/2022 Positive

Overview This medicine was designated as an orphan medicine for the treatment of invasive Scopulariopsis in the European Union on 14 January 2022. This means that the developer will receive scientific and regulatory support from EMA to advance their …

Distribution channels: Healthcare & Pharmaceuticals Industry

Published on April 8, 2024 - 16:03 GMT

Orphan designation: melatonin Treatment of perinatal asphyxia, 02/04/2012 Positive

At the time of designation, perinatal asphyxia affected less than 1 in 10,000 people in the European Union (EU)*. This is equivalent to a total of fewer than 51,000 people, and is below the ceiling for orphan designation, which is 5 people in 10,000. This …

Distribution channels: Healthcare & Pharmaceuticals Industry

Published on April 8, 2024 - 16:03 GMT

Orphan designation: Recombinant adeno-associated viral vector encoding a human micro-dystrophin gene under the control of a muscle ...

At the time of designation, DMD affected less than 0.5 in 10,000 people in the European Union (EU). This was equivalent to fewer than 26,000 people*, and is below the ceiling for orphan designation, which is 5 people in 10,000. This isbased on the …

Distribution channels: Healthcare & Pharmaceuticals Industry

Published on April 8, 2024 - 16:03 GMT

Orphan designation: Adeno-associated virus serotype rh10 containing the human GALC gene Treatment of Krabbe disease, 15/10/2021 ...

Overview This medicine was designated as an orphan medicine for the treatment of Krabbe disease in the European Union on 15 October 2021. This means that the developer will receive scientific and regulatory support from EMA to advance their medicine to …

Distribution channels: Healthcare & Pharmaceuticals Industry

Published on April 8, 2024 - 16:03 GMT

Orphan designation: Nirogacestat Treatment of soft tissue sarcoma, 17/10/2019 Positive

At the time of designation, soft tissue sarcoma affected approximately 4.6 in 10,000 people in the European Union (EU). This was equivalent to a total of around 238,000 people*, and is below the ceiling for orphan designation, which is 5 people in 10,000. …

Distribution channels: Healthcare & Pharmaceuticals Industry

Published on April 8, 2024 - 16:03 GMT

Orphan designation: (S)-2-isobutyrylamino-pentanedioic acid 5-amide…

Overview On 17 October 2019, orphan designation EU/3/19/2203 was granted by the European Commission to MDC RegAffairs GmbH, Germany, for (S)-2-isobutyrylamino-pentanedioic acid 5-amide 1-{[(2S,5S,8S,11R,12S,15S,18S,21R)-2,8-bis-((S)-sec-butyl)-21-hydroxy-5 …

Distribution channels: Healthcare & Pharmaceuticals Industry

Published on April 8, 2024 - 16:03 GMT

Orphan designation: haematopoietic stem cells and blood progenitors umbilical cord-derived expanded with (1R, 4R)-N1-(2-benzyl-7-(...

At the time of designation, approximately 0.7 in 10,000 people received HSCT every year in the European Union (EU). This was equivalent to a total of around 36,000 people*, and is below the ceiling for orphan designation, which is 5 people in 10,000. This …

Distribution channels: Healthcare & Pharmaceuticals Industry

Published on April 8, 2024 - 16:03 GMT

Orphan designation: Glycyl-L-2-methylprolyl-L-glutamic acid Treatment of fragile X syndrome, 28/07/2015 Positive

At the time of designation, fragile X syndrome affected approximately 2.2 in 10,000 people in the European Union (EU). This was equivalent to a total of around 113,000 people*, and is below the ceiling for orphan designation, which is 5 people in 10,000. …

Distribution channels: Healthcare & Pharmaceuticals Industry

Published on April 8, 2024 - 16:03 GMT

Orphan designation: Azithromycin dihydrate Prevention of bronchopulmonary dysplasia, 14/01/2022 Positive

Overview This medicine was designated as an orphan medicine for the prevention of bronchopulmonary dysplasia in the European Union on 14 January 2022. This means that the developer will receive scientific and regulatory support from EMA to advance their …

Distribution channels: Healthcare & Pharmaceuticals Industry

Published on April 8, 2024 - 16:03 GMT

Orphan designation: Allogeneic cultured postnatal thymus-derived tissue Treatment of CHARGE syndrome, 26/02/2019 Positive

CHARGE syndrome is a genetic disorder often caused by mutations (changes) in the genes for a protein called CHD7 that is needed for normal development of an unborn child. Patients with CHARGE syndrome may have an eye defect called coloboma, where part of …

Distribution channels: Healthcare & Pharmaceuticals Industry

Published on April 8, 2024 - 16:03 GMT

Orphan designation: Allogeneic cultured postnatal thymus-derived tissue Treatment of DiGeorge syndrome, 26/02/2019 Positive

The effects of the medicine have been evaluated in experimental models. At the time of submission of the application for orphan designation, clinical trials with the medicine in patients with DiGeorge syndrome who have a non-functional thymus were ongoing …

Distribution channels: Healthcare & Pharmaceuticals Industry

Published on April 8, 2024 - 16:00 GMT

Renowned Divorce Lawyer Timothy Verhaeghe From Verhaeghe Law Office Wins 2024 ThreeBestRatedⓇ Award For Edmonton Region

EDMONTON, ALBERTA, CANADA, April 8, 2024 /⁨EINPresswire.com⁩/ -- Timothy Verhaeghe From Verhaeghe Law Office is felicitated with the 2024 ThreeBestRatedⓇ Award of Excellence for performing as the best divorce lawyer in the region. He is hand picked by the …

Distribution channels: Law ...

Published on April 8, 2024 - 16:00 GMT

Advantage Club launches 1 week onboarding program of rewards & recognition for Blueboard customers affected by shutdown

SAN FRANCISCO, USA, April 8, 2024 /⁨EINPresswire.com⁩/ -- Advantage Club, a leading global employee engagement platform, has launched a special Rewards and Recognition (R&R) program designed specifically for the customers of Blueboard, an employee …

Distribution channels: Business & Economy, Companies ...