RDR Alert®

In this video, Beth Stein, M.D. and myasthenia gravis patient Anaya Mitchell explain how raising awareness is changing the landscape for patients with myasthenia gravis and leading to medical breakthroughs for the patient population.

It was announced this morning that dasiglucagon has met its primary and key secondary objectives in its multinational Phase 3 clinical trial in patients with severe hypoglycemia.

This morning, Cellectar Biosciences announced that the U.S. FDA has granted orphan drug designation to CLR 131 for the treatment of neuroblastoma.

The U.S. FDA granted Fast Track designation to nintedanib for the treatment of systemic sclerosis with associated interstitial lung disease (SSc-ILD).

Phase 2 clinical and immunological data presented at the 44th Annual EBMT Meeting this morning revealed that galinpepimut-S is safe and effective in high-risk multiple myeloma.

At the ENDO Annual Meeting in Chicago, Progenics presented biochemical tumor marker data from its pivotal Phase 2 trial of iobenguane I 131 (Azedra) in patients with pheochromocytoma and paraganglioma

Results from a study published in the Orphanet Journal of Rare Diseases this week suggest that the hereditary neuropathy with liability to pressure palsies, which has been assumed to be rare, might not be uncommon in the Korean population.

On Wednesday, the first patient with primary hyperoxaluria (PH) was randomized into OxThera AB’s Phase 3 trial of OC5 (Oxabact).

Hartmann Wellhoefer, M.D., head of medical affairs at Shire, recently sat down with Rare Disease Report to discuss the company's recent work for the rare disease community, particularly lysosomal storage diseases.

This morning, it was announced that the U.S. FDA has approved CSL Behring’s maintenance therapy for the treatment of patients with chronic inflammatory demyelinating polyneuropathy (CIDP).

ALXN1210 has demonstrated non-inferiority to eculizumab in complement inhibitor treatment-naïve patients with paroxysmal nocturnal hemoglobinuria, according to results from a pivotal Phase 3 study.

The U.S. Food and Drug Administration has granted Fast Track designation to gaboxadol (OV101), a therapy in development for the treatment of Fragile X syndrome.

Abeona announced that the U.S. FDA has granted rare pediatric disease designation to ABO-202, a gene therapy in development for the treatment of infantile and late infantile-onset Batten disease.

The U.S. FDA has approved hydroxycarbamide/hydroxyurea tablets in pediatric patients suffering from sickle cell anemia, ages two years and older, France-based Addmedica has announced.

The U.S. FDA has placed a Clinical Hold on the IGNITE DMD Phase 2/3 trial for SGT-001 microdystrophin gene transfer in Duchenne muscular dystrophy, Solid Biosciences Inc. has announced.

Results from the Phase 3 PERSIST-2 clinical trial of the investigational JAK2 inhibitor pacritinib have been published online in The Journal of the American Medical Association (JAMA) Oncology.

World-renowned Cambridge University physicist and best-selling author Stephen Hawking died at his home in England early this morning. He was 76 years old.

Jupiter Orphan Therapeutics has submitted the first of the several highly anticipated investigational new drug applications for potential therapies that utilize Jotrol, its platform product.

Teva Pharmaceuticals, the HDSA, and the ALS Association are partnering for the Teva CNS Target Identification Challenge, a crowdsourcing effort to seek novel targets with therapeutic potential.

The Medical College of Wisconsin has received clearance from the U.S. FDA for the previously unannounced IND application for the Phase 1 trial of a combination therapy for relapsed or refractory AML.

This morning, Alynylam Pharmaceuticals, Inc. has announced the Bridge the Gap initiative in which it will partner with a family affected by hATTR amyloidosis for a new book. The initiative, called , will allow the family that has lived with the rare disease for generations to share its personal accounts. The book is titled “Living a Rare Life,” and can be found, along with other educational content, at hATTRBridge.com.

Interim clinical data from the ongoing BP-004 trial suggest that BPX-501 reduces cancer recurrence rates in pediatric patients with acute myeloid leukemia and primary immunodeficiencies.

Anaya Mitchell was diagnosed with myasthenia gravis (MG) in the Summer of 2013. She was 13 years old. In this video, describes the challenging road she had on her way to diagnosis.

A February 2018 Type C meeting between Sarepta Therapeutics, Inc. and the Division of Neurology Products of the U.S. FDA was held to solicit the Division’s guidance on the development pathway for Sarepta’s golodirsen (SRP-4053).

On the heels of announcing that the Odyssey Outcomes trial met its primary endpoint, Regeneron and Sanofi are ensuring more affordable and timely access to patients who need alirocumab (Pralutent Injection).