Page 84 - Orphan News Today

NCPCR writes to states, UTs over issue of children orphaned due to COVID-19 ANI | Updated: May 03, 2021 13:17 IST New Delhi [India], May 3 (ANI): National Commission for Protection of Child Rights (NCPCR) has written a letter to the chief secretary of all states and union territories regarding the issue of children who have become orphan due to the death of both parents due to COVID-19. The commission has requested that if any such information for an abando...

MitoChem Therapeutics, Inc.: MitoChem Therapeutics Granted FDA Orphan Drug Designation for Treatment of Retinitis Pigmentosa CHARLESTON, SC / ACCESSWIRE / April 14, 2021 / MitoChem Therapeutics, Inc., a privately held company developing therapies for diseases involving mitochondrial dysfunction, announced today that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation status to its lead compound, MC16, for treatment of retinitis pigmentosa (RP). Retinitis pigmentosa i...

06 aprile 2021 13:40 Fonte: Adnkronos #salute-e-benessere - BEIJING, and BRIDGEWATER, N.J., April 6, 2021 /PRNewswire/ -- Gan & Lee Pharmaceuticals Co., Ltd. (hereinafter referred to as Gan & Lee, stock code: 603087.SH), a global biopharmaceutical company, today announced that the European Medicine Agency (EMA) Committee for Orphan Medicinal Products granted orphan drug designation for the investigational compound GLR2007, for the treatment of glioma. Glioma is a broad term describing neuroe...

Top Story March 17, 2021 ISLAMABAD: Minister for Science and Technology Fawad Chaudhry said on Tuesday the government was working on initiation of contempt of court proceedings against the Election Commission of Pakistan (ECP) if it did not tender resignation. He was briefing the media about the decisions taken at the cabinet meeting here, which met with Prime Minister Imran Khan in the chair after the election of the chairman and deputy chairman of Senate. The minister said consultations were...

(3) Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, today announced that the European Medicines Agencys Committee for Orphan Medicinal Products (COMP) released details supporting the Orphan Designation of BIVV003, an investigational ex vivo gene-edited cell therapy product candidate currently being evaluated for the treatment of sickle cell disease in the Phase 1/2 PRECIZN-1 study partnered with Sanofi. The Committees decision to grant Orphan Designation was based in par...

(1) TOKYO, Mar 12, 2021 - (JCN Newswire) - Eisai Co., Ltd. announced today that it has received orphan drug designation for LENVIMA (generic name: lenvatinib mesylate), the orally available multiple receptor kinase inhibitor discovered by Eisai, with a prospective indication for uterine body cancer, by the Ministry of Health, Labour and Welfare (MHLW). In Japan, the estimated number of patients with uterine body cancer is approximately 30,000.1 It is estimated that in 2020, there were more than...

WASHINGTON (dpa-AFX) - Alkermes plc (ALKS), a global biopharmaceutical company developing medicines in the fields of neuroscience and oncology, said nemvaleukin alfa, its investigational engineered

DUBLIN, March 11, 2021 /PRNewswire/ -- Alkermes plc (Nasdaq: ALKS) today announced that nemvaleukin alfa ("nemvaleukin", formerly referred to as ALKS 4230), the company's investigational engineered

About 15 years ago, Caledonia residents Kam and William Buhler decided they wanted to help people outside of their family — far, far outside of their family. They have two daughters: one who was a teenager and another in her 20s at the time. But they wanted to do something more to help struggling teenagers overseas. In 2004 or 2005, Kam Buhler read a news article about an American who sponsored children in Ukraine. Buhler, who likes to travel but had never been to Ukraine before, wanted to...

PRESS RELEASE March 3, 2021 Saniona (OMX: SANION), a clinical stage biopharmaceutical company focused on rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted