RDR Alert®

First-line avelumab monotherapy treatments show efficacy in patients with Merkel cell carcinoma (mMCC).

AbbVie won’t seek accelerated approval for Rovalpituzumab Tesirine in third-line relapsed/refractory small cell lung cancer due to the low percentages in efficacy in the TRINITY study.

This morning, Protagonist Therapeutics announced that the Phase 2b PROPEL study of PTG-100 in patients with ulcerative colitis has been discontinued.

A recent study suggests low doses of oral cyclosporine A do not prevent second-eye involvement in adult patients with unilateral Leber’s hereditary optic neuropathy (LHON).

Data presented at the 16th International Symposium on Amyloidosis in Japan were presented this morning, showing that inotersen-treated patients with hATTR amyloidosis continued to exhibit sustained benefit.

Data from a Phase 2 study suggests that voxtalisib has a promising safety profile, but might not be as effective in patients with aggressive malignancies as it is in patients with follicular lymphoma.

A Phase 2a clinical study evaluating Molgradex was recently initiated by Savara, Inc. The study intends to evaluate the reduction of sputum samples without growth of non-tuberculosis mycobacteria during the treatment period.

Positive data was anounced regarding PBI-4050, a possible treatment for Alström syndrome.

In February, the U.S. FDA accepted the New Drug Application for migalastat, a potential Fabry disease therapy in development by Amicus. This morning, it was announced that it was approved in Japan.

A team of researchers have found that targeting the MLH3 protein involved in DNA repair can reduce the GAA repeat expansion that results in Friedreich's ataxia

This new infographic from Rare Disease Report provides 5 little-known facts about the Huntington's disease that might not necessarily be covered in news articles.

The U.S. FDA has expanded the approval for nilotinib to include the treatment of first- and second-line pediatric patients with Philadelphia chromosome-positive chronic myeloid leukemia in the chronic phase (Ph+ CML-CP).

A 40-year old patient at the UNC Clinical and Translational Research Center was treated with SB-913 this week.

A genetic mutation that has previously been linked to Parkinson’s disease and Alzheimer’s disease could also be connected to ALS, according to a team of researchers supported by the National Institute of Health.

Positive data from a Phase 3 study of inotersen (IONIS-TTRx) in patients with hereditary ATTR amyloidosis was presented at the 2018 American College of Cardiology Annual Meeting.

The U.S. FDA has granted Pedmark, a unique combination of cisplatin and sodium thiosulfate, Fast Track designation for the prevention of cisplatin-related ototoxicity in pediatric patients with standard risk hepatoblastoma.

In this video, Beth Stein, M.D., and myasthenia gravis patient Anaya Mitchell discuss mestinon, steroids, and other treatment methods for myasthenia gravis.

The U.S. FDA has approved brentuximab vedotin (Adcetris) in combination with chemotherapy for adult patients with previously untreated Stage III or IV classical Hodgkin lymphoma.

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.