Experimental Gene Therapy Allows Kids with Inherited Deafness to Hear
Gene therapy has allowed several children born with inherited deafness to hear.
A small study published Wednesday documents significantly restored hearing in five of six kids treated in China. On Tuesday, the Children’s Hospital of Philadelphia announced similar improvements in an 11-year-old boy treated there. And earlier this month, Chinese researchers published a study showing much the same in two other children.
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So far, the experimental therapies target only one rare condition. But scientists say similar treatments could someday help many mor...
Source: TIME: Health - January 25, 2024 Category: Consumer Health News Authors: LAURA UNGAR/AP Tags: Uncategorized wire Source Type: news
Health Canada approves Pfizer's gene therapy for treatment of hemophilia
Pfizer said on Wednesday that Canada's health regulator approved its gene therapy for the treatment of a rare inherited bleeding disorder called hemophilia B ahead of a U.S. decision. (Source: CBC | Health)
Source: CBC | Health - January 3, 2024 Category: Consumer Health News Tags: News/Health Source Type: news
Gene Therapy for Hemophilia Becomes a Reality
(MedPage Today) -- Earlier this year we reported on FDA's approval of valoctocogene roxaparvovec (Roctavian) for adults with severe hemophilia A. In this story, we provide an update on what has happened since.
Two gene therapies have now been... (Source: MedPage Today Hematology/Oncology)
Source: MedPage Today Hematology/Oncology - December 29, 2023 Category: Hematology Source Type: news
Synergy Billing Corp. Welcomes WellPha Specialty Pharmacy to its Customer Portfolio
Revenue cycle management and consulting firm pairs a national reach with a boutique approach to deliver custom solutions for WellPha in the Medicare hemophilia patient population HIGH POINT, N.C., Dec. 12, 2023 /PRNewswire-PRWeb/ -- Synergy Billing Corp., a boutique consulting and... (Source: PRWeb: Medical Pharmaceuticals)
Source: PRWeb: Medical Pharmaceuticals - December 12, 2023 Category: Pharmaceuticals Tags: LIC Source Type: news
Novel Monoclonal Antibody Reduces Bleeds in Hemophilia A and B
(MedPage Today) -- SAN DIEGO -- The investigational monoclonal antibody marstacimab reduced the rate of treated bleeds in patients with hemophilia A or B without inhibitors to factor VIII or factor IX, the phase III BASIS trial showed.
The annualized... (Source: MedPage Today Hematology/Oncology)
Source: MedPage Today Hematology/Oncology - December 12, 2023 Category: Hematology Source Type: news
FDA and EMA Accept Marstacimab Regulatory Submissions for the Treatment of Hemophilia A and B
Submissions based on positive data from the Phase 3 BASIS trial, which were presented this past weekend at the American Society of Hematology (ASH) Annual Meeting
If approved in the U.S. and EU, marstacimab could become the first once-weekly... (Source: Drugs.com - New Drug Applications)
Source: Drugs.com - New Drug Applications - December 11, 2023 Category: Drugs & Pharmacology Source Type: news
Emicizumab Safe, Effective, in Infants With Hemophilia A
(MedPage Today) -- SAN DIEGO -- Administering emicizumab (Hemlibra) to previously untreated or minimally treated infants with severe hemophilia A without factor VIII inhibitors is effective and safe, according to results from research presented... (Source: MedPage Today Hematology/Oncology)
Source: MedPage Today Hematology/Oncology - December 10, 2023 Category: Hematology Source Type: news
New data reinforce the benefit of early preventative treatment with Roche ’s Hemlibra for babies with severe haemophilia A
Phase III HAVEN 7 primary data presented at ASH 2023 provide additional confidence in the favourable efficacy and safety profile of subcutaneous Hemlibra given soon after birth1At nearly two years median follow-up in the descriptive, single-arm study, no babies experienced spontaneous bleeds requiring treatment, and all treated bleeds were as a result of trauma1Safety results were consistent with previous studies of Hemlibra, with no new safety signals observed1The HAVEN 7 study was developed in collaboration with the haemophilia A community, to generate additional evidence for the prophylactic treatment of infants with ha...
Source: Roche Media News - December 9, 2023 Category: Pharmaceuticals Source Type: news
New data reinforce the benefit of early preventative treatment with Roche ’s Hemlibra for babies with severe haemophilia A
Phase III HAVEN 7 primary data presented at ASH 2023 provide additional confidence in the favourable efficacy and safety profile of subcutaneous Hemlibra given soon after birth1At nearly two years median follow-up in the descriptive, single-arm study, no babies experienced spontaneous bleeds requiring treatment, and all treated bleeds were as a result of trauma1Safety results were consistent with previous studies of Hemlibra, with no new safety signals observed1The HAVEN 7 study was developed in collaboration with the haemophilia A community, to generate additional evidence for the prophylactic treatment of infants with ha...
Source: Roche Investor Update - December 9, 2023 Category: Pharmaceuticals Source Type: news
Palestinian Families Seek Medical Care Outside Besieged Gaza
ARISH, Egypt—Nazha Abu Dhaahir was relieved when her 7-year-old son Yousef was chosen to leave the Gaza Strip for Abu Dhabi for treatment of his hemophilia, which can render any injury fatal. But that meant leaving two sons who have the same blood disorder in a war zone. She and Yousef rushed to…#arish #egypt #nazhaabudhaahir #gazastrip #abudhabi #hemophilia #yousef #gaza #emirati #sinaipeninsula (Source: Reuters: Health)
Source: Reuters: Health - December 8, 2023 Category: Consumer Health News Source Type: news
Contaminated blood scandal victims say Rishi Sunak 'should be ashamed' after PM was 'forced to do right thing' and compensate families quickly following humiliating Tory revolt
The Haemophilia Society said Rishi Sunak 'should be ashamed' that it took political pressure and public opinion to force the UK Government 'to do the right thing'. (Source: the Mail online | Health)
Source: the Mail online | Health - December 5, 2023 Category: Consumer Health News Source Type: news
Sanofi CEO under pressure to woo investors after spending shock
[1/2] Paul Hudson, chief executive officer of Sanofi, speaks during the annual results news conference at the company's headquarters in Paris, France, February 4, 2022. REUTERS/Benoit Tessier/File Photo Acquire Licensing Rights Dec 5 (Reuters) - Sanofi's (SASY.PA) CEO is under pressure to tell…#paulhudson #sanofi #hudson #danlyons #denver #dupixent #haemophilia #altuviiio #beyfortus #tzield (Source: Reuters: Health)
Source: Reuters: Health - December 5, 2023 Category: Consumer Health News Source Type: news
Sernova Receives Orphan Drug and Rare Pediatric Disease Designations for its Hemophilia A Program from FDA
LONDON, Ontario and WINDHAM COUNTY, Conn., Nov. 27, 2023 (GLOBE NEWSWIRE) -- Sernova Corp. SVA SEOVF (FSE/XETRA:PSH), a clinical-stage company and leader in cell therapeutics, today announced the U.S. Food and Drug Administration (FDA) has granted both Orphan Drug Designation (ODD) and Rare…#ontario #windhamcounty #conn #sernovacorp #psh #fda #hemophilia #hemophiliaa #cellpouch #factorviii (Source: Reuters: Health)
Source: Reuters: Health - November 27, 2023 Category: Consumer Health News Source Type: news
EU Approval of AAV5 DetectCDx(TM) Provides Access to Companion Diagnostic for Hemophilia A Gene Therapy
SALT LAKE CITY, Nov. 10, 2023 -- (Healthcare Sales & Marketing Network) -- ARUP Laboratories today announced that it has gained the Conformité Européenne (CE) mark for AAV5 DetectCDx single-site use under the European Union's In Vitro Diagn... Diagnostics, Regulatory ARUP Laboratories, BioMarin Pharmaceutical, AAV5 DetectCDx, hemophilia A (Source: HSMN NewsFeed)
Source: HSMN NewsFeed - November 10, 2023 Category: Pharmaceuticals Source Type: news
Why BioMarin Pharmaceutical (BMRN) Stock Is Trading Higher Today
Activist investor Elliott Investment Management has taken a significant position in BioMarin Pharmaceutical Inc BMRN, engaging with the company on its strategic direction amid leadership changes and the evolving status of its hemophilia treatment, Roctavian. The hedge fund has invested over $1…#activist #bmrn #hemophilia #biomarin #roctavian #isharesbiotechnology #ibb #medicare #jeanjacquesbienaime #alexanderhardy (Source: Reuters: Health)
Source: Reuters: Health - November 7, 2023 Category: Consumer Health News Source Type: news
