Results 1 to 10 of about 1,478,682 (342)

Safety of Onasemnogene Abeparvovec for Patients With Spinal Muscular Atrophy 8.5 kg or Heavier in a Global Managed Access Program

Pediatric Neurology, 2022
Spinal muscular atrophy is a rare, neurodegenerative disorder caused by biallelic deletions in the survival motor neuron (SMN1) gene. Onasemnogene abeparvovec is a one-time, intravenous gene replacement therapy designed to deliver the SMN1 transgene. Although available in many geographies, it is not approved globally. The Global Managed Access Program (
Deepa H Chand, Sandra P Reyna
exaly   +5 more sources

Global Expanded Access Program for Pemigatinib in Patients with Previously Treated Locally Advanced or Metastatic Cholangiocarcinoma and Fibroblast Growth Factor Receptor Gene Alterations

Cancer Research and Treatment
Purpose Pemigatinib is a fibroblast growth factor receptor-2 (FGFR2) inhibitor approved for use in patients with previously treated cholangiocarcinoma (CCA) and FGFR2 fusions or rearrangements. This ongoing global Expanded Access Program (EAP) allows physicians in regions where pemigatinib is not commercially available to request pemigatinib for ...
Anouk Lindley, Gerhard Prager, Michael Bitzer   +2 more
exaly   +5 more sources

Efficacy and safety of avelumab treatment in patients with metastatic Merkel cell carcinoma: experience from a global expanded access program [PDF]

Journal for ImmunoTherapy of Cancer, 2020
Background Avelumab, a human anti–programmed death-ligand 1 immunoglobulin G1 monoclonal antibody, showed favorable efficacy and safety in patients with metastatic Merkel cell carcinoma (mMCC) in the phase II JAVELIN Merkel 200 trial, leading to approval in multiple countries.
John W Walker, Celeste Lebbé, Giovanni Grignani, Paul Nathan, Luc Dirix, Eyal Fenig, Paolo Antonio Ascierto, Shahneen Sandhu, Rodrigo Munhoz, Elena Benincasa, Sarah Flaskett, Josh Reed, Arne Engelsberg, Subramanian Hariharan, Vijay Kasturi   +14 more
openaire   +7 more sources

Improving Global Access to Transgender Health Care: Outcomes of a Telehealth Quality Improvement Study for the Air Force Transgender Program

Transgender Health, 2022
Purpose: To describe the development of the United States Air Force's (USAF) telehealth program from fall 2017 through fall of 2020 in response to the unique challenges associated with providing care for a global transgender military population. Methods:
Joshua M, Smalley, Janelle M, Lozano, Chari' J, McMahon, Jeffrey A, Colburn   +3 more
openaire   +3 more sources

Innovation intermediaries as agents for SMEs' organisational learning: a case study on the UCLA's global access program

International Journal of Technology Intelligence and Planning, 2015
Research on open innovation has not yet built a comprehensive conceptualisation on how innovation intermediaries help small and medium enterprises (SMEs) in their capability development. In this paper we analyse the role that innovation intermediaries play in supporting SMEs in their international growth.
NEIROTTI, Paolo, PAOLUCCI, EMILIO
openaire   +4 more sources

High response rate to bortezomib with or without dexamethasone in patients with relapsed or refractory multiple myeloma: results of a global phase 3b expanded access program [PDF]

British Journal of Haematology, 2008
SummaryPhase 2 trials have demonstrated that bortezomib ± dexamethasone is safe and effective in relapsed multiple myeloma (MM). In this multicentre, open‐label, phase 3b trial, 638 patients with relapsed or refractory MM (median 3 prior therapies) received bortezomib 1·3 mg/m2 on days 1, 4, 8, and 11 of a maximum of eight 3‐week cycles (median 5 ...
Mikhael JR, Belch AR, Prince HM, Lucio MN, Maiolino A, Corso A, Petrucci MT, Musto P, Komarnicki M, Stewart AK   +9 more
openaire   +3 more sources

Implementing a Global Expanded Access Program (EAP) for Infantile-Onset Spinal Muscular Atrophy (Type I): Understanding the Imperative, Impact and Challenges

Journal of Neuromuscular Diseases, 2019
Nusinersen is the first disease-modifying therapy approved for the treatment of spinal muscular atrophy (SMA), a rare genetic disorder characterized by severe progressive muscular atrophy and weakness. An expanded access program (EAP) provides investigational treatment to patients without other treatment options.
Yong, Jonathan, Moffett, Megan, Lucas, Sam   +2 more
openaire   +4 more sources

DataCite Global Access Program

, 2023
A presentation about DataCite's Global Access Program (GAP) presented at DataCite's community meeting, Open Hours.
, Gabi, , Bosun, , Arturo
openaire   +4 more sources

Drug and Therapeutics Committee (DTC) evolvement and expanded scope in Ethiopia [version 2; peer review: 2 approved]

Gates Open Research, 2023
As a key partner of Ministry of Health (MOH) Ethiopia, The Clinton Health Access Initiative (CHAI) had been implementing the Child Survival Project (CSP) since October 2015.
Dinkineh Bikila, Zinabie Feleke, Seid Ali, Alebel Yaregal, Audrey Battu, Amsalu Demisie, Regasa Bayisa, Felix Lam, Salem Fisseha, Habtamu Seyoum, Yigeremu Abebe   +10 more
doaj   +1 more source

Human papillomavirus testing using existing nucleic acid testing platforms to screen women for cervical cancer: implementation studies from five sub-Saharan African countries

BMJ Open, 2023
Objectives To demonstrate acceptability and operational feasibility of introducing human papillomavirus (HPV) testing as a principal cervical cancer screening method in public health programmes in sub-Saharan Africa.Setting 45 primary and secondary ...
Trevor Peter, Jessica Joseph, Bernard Madzima, Mamadou Diop, Susan Nabadda, Owen Demke, Shaukat Khan, Lola Ameyan, Joseph Bitilinyu-Bango, Blandine Bourgoin, Babacar Guèye, Jibrin Kama, Marvin Lubega, Tatenda Maparo, Tasimba Mhizha, Andrew Musoke, Twambilire Phiri, Timothy Tchereni, Owens Wiwa, Karen Hariharan   +19 more
doaj   +1 more source