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Using CRISPR to introduce beneficial but rare gene mutations in sickle cell

People who suffer from the inherited disease sickle cell disease have defects in their hemoglobin, the blood molecule that transports vital oxygen throughout the body. But a small percentage of patients with the disease have beneficial gene mutations that lessen their symptoms. The mutations switch on a gene that produces fetal hemoglobin—a type of the blood molecule that the body normally stops producing in infancy. The lifelong production of fetal hemoglobin helps shield those sickle cell patients from the worst effects of the disease.
- Source: fiercebiotech
- 493
- April 5, 2018
Researches sickle cell disease
EU reviews for Sanofi’s Dupixent, cemiplimab

European regulators have agreed to review Sanofi and Regeneron’s Dupixent for asthma and cemiplimab for advanced cutaneous squamous cell carcinoma.
- Source: pharmatimes
- 1,008
- April 4, 2018
asthma FDA New Approvals Sanofi
Partner Therapeutics (PTx) Acquires Leukine® from Sanofi

Purchase includes Lynnwood, WA Biologics Manufacturing Facility
- Source: finance.yahoo
- 659
- April 4, 2018
acute myelogenous leukemia FDA Leukine Sanofi
Martin Pharmaceuticals Receives FDA Orphan Drug Designation For LIVANTRA™

MARTIN PHARMACEUTICALS RECEIVES FDA ORPHAN DRUG DESIGNATION FOR LIVANTRA™
- Source: martinpharma
- 634
- April 4, 2018
FDA LIVANTRA™ rare (orphan) diseases
Eisai Submits Supplemental New Drug Application (sNDA) to FDA for FYCOMPA® (perampanel) Pediatric Indications

Eisai Inc. announced that it has submitted a supplemental New Drug Application (sNDA) for priority review to the U.S. Food and Drug Administration (FDA) for its antiepileptic drug FYCOMPA® (perampanel) CIII as monotherapy and adjunctive use for the treatment of partial-onset seizures (POS) with or without secondarily generalized seizures in pediatric patients (ages 2 to less than 12 years). The submission also proposes a pediatric indication for monotherapy and adjunctive use for primary generalized tonic-clonic seizures (PGTC) in children (ages 2 to less than 12 years) with epilepsy. The sNDA is for both the FYCOMPA tablet and oral suspension formulations.
- Source: prnewswire
- 661
- April 4, 2018
Eisai FDA FYCOMPA® New Approvals
On-demand health concierge Accolade lands another $50M in funding

Last week on-demand healthcare concierge for provider employers Accolade received $50 million in funding led by Andreessen Horowitz, Carrick Capital Partners, Madrona Venture Group, and McKesson Ventures, with participation from Cross Creek Advisors and Madera Technologies.
- Source: MobiHealthNews
- 760
- April 4, 2018
Company Insights funding healthcare
Study suggests link between anxiety and smartphone addiction

Feeling emotionally unstable could lead you to rely on your smartphone more, according to new research.
- Source: CTVNews
- 671
- April 4, 2018
Mental health mHealth Researches
Tele-rehabilitiaion platform TheraNow launches

Folks in need of physical therapy services may not have to leave the house for treatment. Therainc has just launched its latest product, TheraNow, an online platform that connects patients to physical therapists through teletherapy.
- Source: MobiHealthNews
- 743
- April 4, 2018
EHR New Approvals Telemedicine
Another immunometabolism deal? Johns Hopkins spinout Dracen lands $36M investment from Deerfield

The company, founded just a year ago, is developing novel glutamine antagonists that work as inhibitors of cancer metabolism, choking out tumors and their normal metabolic function.
- Source: Endpts
- 615
- April 3, 2018
cancer inhibitor Market Views metabolism
UCB Announces Filing Acceptance with the China Food and Drug Administration for CIMZIA® (certolizumab pegol) for the Treatment of Moderate-to-Severe Rheumatoid Arthritis

UCB, a global biopharmaceutical company, announced the submission of an application for an Import Drug License to the Chinese Food and Drug Administration (CFDA) for the approval of CIMZIA® (certolizumab pegol) to treat moderate-to-severe rheumatoid arthritis (RA).
- Source: ucb
- 790
- April 3, 2018
CFDA CIMZIA® New Approvals RA

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