DMD Gene Therapy Safe, Effective at 4 Years DMD Gene Therapy Safe, Effective at 4 Years
Four years after a single dose of SRP-9001 gene therapy, four children with Duchenne muscular dystrophy show sustained stabilization of motor function that would otherwise show expected decline.Medscape Medical News (Source: Medscape Neurology and Neurosurgery Headlines)
Source: Medscape Neurology and Neurosurgery Headlines - November 6, 2023 Category: Neurology Tags: Neurology & Neurosurgery News Source Type: news
Gene Therapy Controls Diabetic Eye Disease for at Least a Year
(MedPage Today) -- SAN FRANCISCO -- A form of gene therapy stabilized or improved diabetic retinopathy (DR) for up to a year, particularly in patients with nonproliferative (NP) DR, a small clinical study showed.
Overall, about 40% of patients... (Source: MedPage Today Endocrinology)
Source: MedPage Today Endocrinology - November 6, 2023 Category: Endocrinology Source Type: news
For A Change There ’s Optimism About A Gene Therapy’s Commercialization—Vyjuvek For Wound Care
Cantor Fitzgerald said it expects a “very strong product launch” of a gene therapy made by Krystal Biotech, the topical gel Vyjuvek which treats a rare skin disease. (Source: Forbes.com Healthcare News)
Source: Forbes.com Healthcare News - November 6, 2023 Category: Pharmaceuticals Authors: Joshua Cohen, Senior Contributor Tags: Healthcare /healthcare Innovation /innovation business pharma & standard Source Type: news
QuickSTAT recognized as the best cell and gene therapy logistics provider in the Asia-pacific region by ACGTEA
Award honors exceptional performance from providers that facilitate the advancement of new therapies, R&D and manufacturing. NEW YORK, Nov. 5, 2023 /PRNewswire-PRWeb/ -- For the second consecutive year, QuickSTAT has been recognized by the Asia-Pacific Cell & Gene Excellence Awards... (Source: PRWeb: Medical Pharmaceuticals)
Source: PRWeb: Medical Pharmaceuticals - November 5, 2023 Category: Pharmaceuticals Tags: AWD Source Type: news
Gene Therapy Market size to grow by USD 4.61 billion from 2022 to 2027, North America to account for 39% of market growth- Technavio
NEW YORK, Nov. 3, 2023 /PRNewswire/ -- The gene therapy market size is expected to grow by USD 4.61 billion from 2022 to 2027. In addition, the momentum of the market will progress at a CAGR of 19.91% during the forecast period, according to Technavio Research. The market has been... (Source: PRWeb: Medical Pharmaceuticals)
Source: PRWeb: Medical Pharmaceuticals - November 3, 2023 Category: Pharmaceuticals Tags: MRR Source Type: news
The Eyes Have It: Gene Therapy Offers Hope For Glaucoma Treatment
According to the World Health Organization (WHO), glaucoma is the second leading cause of blindness globally. (Source: Forbes.com Healthcare News)
Source: Forbes.com Healthcare News - November 3, 2023 Category: Pharmaceuticals Authors: William A. Haseltine, Contributor Tags: Healthcare /healthcare Innovation /innovation business pharma & standard Source Type: news
15th Annual SCOPE: Summit for Clinical Ops Executives Puts Spotlight on the Cell & amp; Gene Therapies Supply Chain & amp; Medical Device Trials, February 11-14, 2024, Orlando, FL
Over 3,300 executives will convene to explore solutions for various aspects of planning and managing clinical trials. NEEDHAM, Mass., Nov. 2, 2023 /PRNewswire-PRWeb/ -- In response to the rapid expansion of the cell and gene therapy (CGT) market valued at $7.28 billion in 2022 and a... (Source: PRWeb: Medical Pharmaceuticals)
Source: PRWeb: Medical Pharmaceuticals - November 2, 2023 Category: Pharmaceuticals Tags: TDS Source Type: news
Two Sickle Cell Disease Gene Therapies May Soon Be Approved By FDA
FDA will soon decide approval of exa-cel and lovo-cel for sickle cell disease. These may be one-time treatment options for SCD patients burdened by unmet needs. (Source: Forbes.com Healthcare News)
Source: Forbes.com Healthcare News - November 2, 2023 Category: Pharmaceuticals Authors: Joshua Cohen, Senior Contributor Tags: Healthcare /healthcare Innovation /innovation business pharma & standard Source Type: news
FDA Advisors Say New Gene Therapy for Sickle Cell Disease Is Safe
WEDNESDAY, Nov. 1, 2023 -- A new gene therapy for sickle cell disease was deemed safe by a U.S. Food and Drug Administration advisory panel on Tuesday, paving the way for full approval by early December.The FDA had already decided that the therapy,... (Source: Drugs.com - Pharma News)
Source: Drugs.com - Pharma News - November 1, 2023 Category: Pharmaceuticals Source Type: news
New Gene Therapy for Sickle Cell Disease Considered Safe, FDA
WEDNESDAY, Nov. 1, 2023 -- A new gene therapy for sickle cell disease was deemed safe by a U.S. Food and Drug Administration advisory panel on Tuesday, paving the way for full approval by early December.
The FDA had already decided that the... (Source: Drugs.com - Pharma News)
Source: Drugs.com - Pharma News - November 1, 2023 Category: Pharmaceuticals Source Type: news
Assess Risks of Gene Therapy in Follow-Up Study: FDA Panel Assess Risks of Gene Therapy in Follow-Up Study: FDA Panel
A panel of advisers to the regulator said on Tuesday Vertex Pharmaceuticals and CRISPR Therapeutics could assess potential safety risks of their sickle cell disease gene therapy after approval.Reuters Health Information (Source: Medscape Medical News Headlines)
Source: Medscape Medical News Headlines - November 1, 2023 Category: Consumer Health News Tags: Hematology-Oncology News Source Type: news
FDA Advisors Say New Gene Therapy for Sickle Cell Disease, Exa-cel, is Safe
WEDNESDAY, Nov. 1, 2023 -- A new gene therapy for sickle cell disease was deemed safe by a U.S. Food and Drug Administration advisory panel on Tuesday, paving the way for full approval by early December.
The FDA had already decided that the... (Source: Drugs.com - Daily MedNews)
Source: Drugs.com - Daily MedNews - November 1, 2023 Category: General Medicine Source Type: news
Potential Cure for Sickle Cell Clears Hurdle for Approval
(MedPage Today) -- An FDA advisory committee meeting on Tuesday did nothing to dispel expectations that the investigational agent exagamglogene autotemcel (exa-cel) will become the first approved gene therapy for sickle cell disease (SCD).
Members... (Source: MedPage Today Public Health)
Source: MedPage Today Public Health - November 1, 2023 Category: American Health Source Type: news
WATCH LIVE: Sickle cell gene therapy gets review from FDA advisory committee
The only cure for painful sickle cell disease today is a bone marrow transplant. But soon there may be a new cure that attacks the disorder at its genetic source. On Tuesday, advisers to the Food and Drug Administration will review a gene therapy for the inherited blood disorder, which in the U.S.…#fda #crispr #nobelprize #allisonking #stlouis #africa #african #middleeastern #crisprtherapeutics #vertex (Source: Reuters: Health)
Source: Reuters: Health - October 31, 2023 Category: Consumer Health News Source Type: news
[Ad hoc announcement pursuant to Art. 53 LR] Roche announces EMBARK trial in Duchenne muscular dystrophy (DMD) did not reach primary endpoint, but shows positive efficacy outcomes on all timed functional key endpoints
This study is ongoing.Study 301 (EMBARK), a Phase 3 global, randomised, double-blinded and placebo-controlled study of Elevidys in ambulatory Duchenne patients aged 4-<8 years old.The ENVOL trial (Study 302) a Phase 2 study in children with Duchenne. The study aims to enrol 21 participants who are under 4 years of age, including newborns. Not yet started.The ENVISION trial (Study 303), a Phase 3 study in older ambulatory/non-ambulatory patients which is now recruiting.The EXPEDITION long-term (5 year) follow up study (Study 305) of participants who have received Elevidys in a previous clinical study, which is not yet re...
Source: Roche Media News - October 31, 2023 Category: Pharmaceuticals Source Type: news
