Rocket Pharmaceuticals, Inc. ("Rocket") has dedicated its mission to developing gene therapy solutions for rare childhood conditions. Recently, the company shared the publication of preliminary data from a Phase…
Emflaza (deflazacort), a corticosteroid, was originally approved by the FDA in 2017 to treat Duchenne muscular dystrophy (DMD) in patients five years or older. This approval was expanded in 2019…
In a recent press release, biopharmaceutical company Aeterna Zentaris Inc. shared that the company would be developing a new therapy for patients with hypoparathyroidism. The preclinical development program will focus…
The majority of Parkinson’s patients treated with Levodopa experience relief from the major motor effects of Parkinson’s. However, according to a recent article in Biospace, after prolonged use of Levodopa…
According to a story from Charcot-Marie-Tooth News, a recent study has demonstrated the potential impacts of an RNA-based therapy as a treatment for type 1A Charcot-Marie-Tooth disease. The treatment was…
In March 2021, the FDA expanded access approval to biotechnology company Longeveron Inc.'s investigational Lomecel-B to a pediatric patient with hypoplastic left heart syndrome (HLHS). According to a press…
Newron Pharmaceuticals and Zambon have entered into an agreement that has the potential to produce a label extension for a treatment for Parkinson's. The agreement is focused on safinamide as…
As reported in NewsWise; rare drugs are few and far between, with a cliental of less than 200,000 Americans dissuading drug companies from taking on the enormous charge of developing…
Since its conception, biotechnology company Chinook Therapeutics, Inc. ("Chinook") has worked to develop therapeutic solutions for patients with kidney diseases. Now, the company enrolled the first patient in a…
During the drug development process, multiple studies are performed to evaluate efficacy, safety, tolerability, pharmacokinetics, and other factors. One of these studies is called a multiple ascending dose study, during which…
For nearly 130 years, pharmaceutical company Merck & Co ("Merck" or "MSD") has been working to develop unique therapeutic solutions for those with rare cancers and a variety of…
The French National Agency for Medicines and Health Products Safety, also known as ANSM, has granted Temporary Authorization of Use of berotralstat for those living with hereditary angioedema (HAE) above…
In mid-March 2021, Mitsubishi Tanabe Pharma Canada ("MTP-CA") shared that its infusion therapy RADICAVA (edaravone) is now available for some Canadian patients with amyotrophic lateral sclerosis (ALS). The treatment will…
We seldom see articles about multiple system atrophy (MSA also called Shy-Drager syndrome). The disease is extremely rare and currently, there is no effective treatment. But according to a recent…
According to a recent article in the Seattle Cancer Care Alliance, the FDA’s recent approval of Axi-cel (Yescarta) will improve the chances of survival for relapsed or refractory follicular lymphoma.…
On March 11, 2021, Patient Worthy attended an educational seminar hosted by the Cleveland Clinic titled Neuromyelitis Optica Spectrum Disorder (NMOSD): Educational Seminar for Patients, Family Members and Caregivers Focus…
Molecular Targeting Technologies (MTTI), a radiopharmaceutical company, announced through Biospace that the FDA has given its approval for MTTI to move forward with a Phase 1 clinical trial to study…
Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…
Patients with hereditary angioedema (HAE) experience recurrent "attacks," or periods of swelling under the skin. Now, in a Phase 2 clinical trial, researchers are evaluating PHVS416 for patients with…
Congress has recently introduced a new bill that would have a significant impact on the rare disease community if it passes. The legislation, called the Speeding Therapy Access Today (STAT)…
A report, commissioned by the National Organization for Rare Disorders and released by IQVIA, has revealed that the majority of orphan drugs, 79%, treat only rare diseases. Despite this fact,…
It is estimated that one person in forty to sixty thousand is affected by Fabry disease. Approximately five hundred Canadians are known to have Fabry disease. Newswise recently carried the…
Biopharmaceutical company OncoSynergy recently shared that the first patient was dosed in a Phase 1 clinical trial. Within this trial, researchers are exploring OS2966 for patients with recurrent glioblastoma.…
According to a story from bloomberg.com, the biopharmaceutical company Rallybio has announced two development programs for therapeutics intended to treat rare diseases. The two programs consist of RLBY212, which is…
At the end of February 2021, BridgeBio Pharma Inc. ("BridgeBio") and its affiliate Origin Biosciences, Inc. ("Origin") shared that its treatment, NULIBRY (fosdenopterin) for Injection, received FDA approval for patients…
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