RDR Alert®

As the Myanmar military’s genocidal actions continue to displace Rohingya refugees, the persecuted minority group is experiencing the largest diphtheria outbreak since the 1920s.

This morning, Abeona Therapeutics announced that the U.S. Food and Drug Administration granted EB-101, its gene therapy for epidermolysis bullosa, Regenerative Medicine Advanced Therapy designation.

This morning, Aradigm announced that it received a Complete Response Letter, denying its New Drug Application for Linhaliq.

Alnylam announced that its Marketing Authorisation Application for patisiran was accepted by the European Medicines Agency.

Novartis has received U.S. FDA approval for lutetium (177Lu) oxodotreotide (Lutathera) for the treatment of somatostatin receptor positive GEP-NETs.

New data concludes that neurofeedback training could potentially increase brain connectivity in patients with Huntington’s disease (HD).

The latest #ALSPepperChallenge video comes from Rare Disease Report. Participate today to raise awareness for ALS.

Rare Disease Report sat down with acute intermittent porphyria patient Nichol Kirby who was diagnosed in 2015 and accused of being a drug seeker looking to feed an opioid addiction.

The peer-reviewed clinical journal Neurology has published the Phase 2 trial results for arimoclomol in patients with SOD1 amyotrophic lateral sclerosis (ALS).

Parent Project Muscular Dystrophy announced that the first patient with Duchenne muscular dystrophy has been dosed with microdystrophin gene therapy.

A study published this week in the Orphanet Journal of Rare Diseases confirmed the importance of genetic findings in the diagnosis of Marfan syndrome.

Faron Pharmaceuticals announced this morning that the U.S. FDA has approved the Investigational New Drug application for Traumakine (FP-1201-lyo) for the treatment of Acute Respiratory Distress Syndrome.

The ipsogen JAK2 RGQ PCR Kit received clearance from the U.S. FDA for additional use in the diagnosis of all myeloproliferative neoplasms.

BERG announced this morning that the U.S. FDA has granted orphan drug designation to the company’s leading product candidate BPM31510 (ubidecarenone) for the treatment of pancreatic cancer.

This morning, Sobi, announced that the FDA has issued a Study may proceed letter for the first study in humans, thereby accepting the investigational new drug application for sobi003, and the regulatory agency granted Fast Track status to the product candidate.

Marshall Summar, M.D., director of the Children’s National Rare Disease Institute, sat down with Rare Disease Report to discuss recent news and what it means for the rare disease community.

Regeneron has announced that Dupixent, a therapy for adult atopic dermatitis (AD) that is inadequately controlled by existing medications, has been granted marketing and manufacturing authorization in Japan.

To kick off the new year, Alnylam Pharmaceuticals, Inc., a company dedicated to the development of RNAi therapeutics, announced the expansion of its Alnylam Act.

The U.S. FDA approved the company’s Investigational New Drug application for initiation of two Phase 3 clinical trials of dasiglucagon in treating congenital hyperinsulinism.

uniQure was granted Orphan Medicinal Product Designation from the European Medicines Agency for AMT-130, an investigational gene therapy for the treatment of Huntington’s disease.

Enzychem Lifesciences has announced that EC-18, a potential therapy for Acute Radiation Syndrome, has been granted Orphan Drug Designation by the U.S. Food and Drug Administration.

Engineers from the Engineering Faculty at the University of Sheffield and Boston Children’s Hospital, Harvard Medical School have created a robot with the intention of esophageal atresia .

Arimoclomol, in development by Danish biotech Orphazyme A/S for the treatment of Niemann-Pick Disease Type C, was granted rare pediatric disease designation by the U.S. FDA this morning.

New research published in Annals of Internal Medicine shows that the rate of arterial and venous thrombosis was significant in patients with myeloproliferative neoplasms (MPNs).

In 2018, uniQure N.V. expects to advance the clinical development of AMT-130, its investigational gene therapy for the treatment of Huntington’s disease.