Mathew Shanley

On Monday, the FDA approved tisagenlecleucel (Kymriah) for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma who have relapsed or are ineligible for an autologous stem cell transplant (ASCT).

Orchard Therapeutics announced that its gene therapy candidate, OTL-200, has been granted Rare Pediatric Disease designation for the treatment of metachromatic leukodystrophy.

Akcea Therapeutics announced that it has expanded its global early access program to the US for its drug volanesorsen, which is in development for treatment of the rare lipid disorder FCS.

REGENXBIO Inc. announced that the U.S. FDA has granted Fast Track designation to RGX-131, a novel, one-time investigational treatment for mucopolysaccharidosis type II (MPS II).

A Rare Pediatric Disease designation was granted by the U.S. FDA this morning to Cellectar Biosciences for CLR 131, intended for the treatment of neuroblastoma.

As stated in article published in the clinical journal Neurobiology of Aging, a team of researchers has discovered that elevated levels of certain types of lipids in the brain could be an early presentation of Parkinson’s disease.

This morning, Acceleron Pharma announced that the US FDA has granted Fast Track designation to ACE-083 for the treatment of patients with facioscapulohumeral muscular dystrophy (FSHD).

This morning, it was announced that the U.S. FDA has approved dabrafenib (Tafinlar) in combination with trametinib (Mekinist) for the adjuvant treatment of patients with melanoma with BRAF V600E or V600K mutation.

Positive results from the Fostamatinib in Thrombocytopenia (FIT) Phase 3 clinical program of fostamatinib disodium hexahydrate (Tavalisse) were published in the American Journal of Hematology this week.

At the Clinical immunology Society Annual Meeting in Toronto on April 27-28, Prometic presented data from its pivotal Intravenous Immunoglobin (IVIG) Phase 3 clinical trial.

The U.S. FDA has accepted for priority review the Biologics License Application for cemiplimab (REGN2810), an investigational anti-PD-1 monoclonal antibody for patients with metastatic CSCC.

This morning, Capricor Therapeutics, Inc. announced that the HOPE-2 clinical trial has been initiated at UC Davis Medical Center in Sacramento, CA.

Results from a questionnaire show that individuals living with FCS prove that communication among people in similar situations can improve a patient’s quality of life.

At the NLA Scientific Sessions, Akcea Therapeutics presented a poster with data from 2 different studies, each of which exhibited a significant reduction of triglycerides in patients with FCS when administered volanesorsen.

At the NLA Scientific Sessions in Las Vegas this morning, Akcea Therapeutics, presented a poster with data from the largest burden of illness study in patients with FCS to date.

At the NLA Scientific Sessions in Las Vegas, NV this weekend, Akcea Therapeutics will present clinical data on volanesorsen and FCS.

This morning, biotechnological company Saniona announced that it has obtained approval to initiate the second part of its Phase 2a trial for Tesomet in adolescents with Prader-Willi syndrome (PWS).

This morning, top-line data from a confirmatory Phase 2 study of NP001 in ALS were reported, and showed that the trial did not meet its primary or secondary endpoints.

IONIS-HTT data shows link between reduction in Huntingtin protein and clinical improvement.

This morning, Lin BioScience announced that the US FDA has granted orphan drug designation to LBS-007 for the treatment of acute lymphoblastic leukemia (ALL).

An observational study in Dutch CAPS patients was conducted by Catharina M. Mulders-Manders and colleagues, and results were published in the Orphanet Journal for Rare Diseases.

At the 70th Annual AAN 2018 Annual Meeting, Alnylam announced new results from the APOLLO Phase 3 study of patisiran, an investigational RNAi therapeutic for the treatment of hATTR amyloidosis.

A result of cooperation between Amerigen Pharmaceuticals and Dipharma, this is the first approved generic equivalent to Actelion Pharmaceuticals’ version of the drug, Zavesca.

The U.S. FDA has cleared the Investigational New Drug application for DTX401 for the treatment of glycogen storage disease type Ia (GSDIa).

Final approval was given this morning to an Abbreviated New Drug Application for Miglustat 100 mg capsules for the treatment of adult patients with mild to moderate type 1 Gaucher disease.

ABO-102, which has previously received rare pediatric disease designation, fast track designation, and orphan drug designation from the U.S. FDA, was the recipient of another acknowledgement this morning when it was granted RMAT designation.

At the 70th AAN Annual Meeting this morning Summit Therapeutics plc presented new 24-week interim data from PhaseOut DMD, its Phase 2 trial of ezutromid in Duchenne.

Researchers from GlaxoSmithKline will collect data from a group of young children treated with Strimvelis in Milan.

On Wednesday, it was announced that Synlogic had initiated and dosed the first subject in its Phase 1/2a study of SYNB1618 for the treatment of phenylketonuria.

According to data presented at the Annual Neuromuscular Translational Research Conference, researchers found that repeat dosing of CAP-1002 results in increased exercise performance in a disease model of Duchenne muscular dystrophy.