RDR Alert®

FLT3mut+ Relapsed/Refractory AML treatment submits New Drug Application.

Potential PKU treatment receives FDA Fast Track designation.

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).

Progeria treatment extends survival in children.

Nusinersen treatment proven to be efficient in spinal muscular atrophy (SMA).

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.

AbobotulinumtoxinA proves effective across multiple therapeutic uses.

New data proves efficacy of relapsing multiple sclerosis (RMS) treatment.

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.

Dr Ali Fatemi, MD, of Kennedy Krieger Institute and John Hopkins University, describes the “Awesome Disease” (LBSL) and how a young girl now inspires his research.

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.

The FDA approved fostamatinib disodium hexahydrate (Tavalisse) tablets for the treatment of thrombocytopenia in adult patients with chronic immune thrombocytopenia. RDR discusses with Dr James Bussel.

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.

The first patient was enrolled in a Phase 3 clinical trial of amifampridine phosphate (Firdapse) in patients with mmuscle-specific kinase (MuSK) antibody positive myasthenia gravis (MuSK-MG).

Dystrophic epidermolysis bullosa (DEB) treatment granted OMPD in Europe.

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.

At the most recent American Lung Association LUNGFORCE expo, RDR sat down with Carly Ornstein to discuss the new resources made available by the ALA to individuals affected by lung fibrosis.

The first patient was dosed in X-linked retinitis pigmentosa (XLRP) treatment.

Positive Phase 1b/2a trial results for RT001 in patients with Friedreich’s ataxia were published this morning in the online journal Movement Disorders.

This morning, the FDA granted Orphan Drug Designation to KL1333 for the treatment of inherited mitochondrial respiratory chain diseases (MRCD).

A survey recently conducted among adults with hereditary spastic paraparesis showed that constipation, alternate constipation and diarrhea, fecal incontinence, and voiding dysfunction are among the considerable problems faced by the patient population.