tag rare-disease-research 
Pharmaceutical Technology
JUNE 29, 2022
This year has already been eventful when it comes to the development of therapies for rare diseases. Additionally, pricing and access for rare disease therapies continue to be scrutinized closely. Prior to the program, only 10 drugs were approved for a rare disease.
Pharmaceutical Technology
JULY 14, 2022
It is estimated that there are currently more than 7,000 orphan diseases, many of which are considered life-threatening and most of which have a genetic basis. Despite this high number, orphan diseases are rare by definition, affecting around one in 2,000 people as defined by the European Union. Go-to-market strategies.
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XTalks
JUNE 28, 2023
Gene therapies for Duchenne muscular dystrophy (DMD) have been an area of intense research and Sarepta’s Elevidys is now the first one to be approved by the US Food and Drug Administration (FDA). DMD is a rare genetic disorder that leads to progressive muscle degeneration and weakness. It is injected into a patient’s muscle tissue.
pharmaphorum
JUNE 2, 2022
R&D into orphan drugs is growing alongside the number of approved treatments, providing treatments for rare diseases that previously did not have any. As covered in a previous article , the importance of R&D to develop treatments for rare diseases is high. of the global population are affected by a rare disease.
XTalks
JANUARY 28, 2022
Uveal melanoma is a rare and aggressive type of melanoma of the eye. Up to 50 percent of patients will develop metastatic disease, mainly spreading to the liver, making treatment difficult and limited. The new treatment will thus help address the unmet need for the disease. The median treatment time is about 23 weeks (or 5.3
pharmaphorum
APRIL 5, 2022
Uveal melanoma is a rare cancer of the eye, which in the majority of patients is localised. mUM is the most common form of cancer affecting the eye in adults, but is still rare, diagnosed in a around 1,700 patients each year in the US, with a few hundred already with metastatic disease that has spread to other tissues.
XTalks
JUNE 8, 2021
In addition, recent data from a Phase I trial has shown ‘remarkable’ long-term results of the gene therapy in children with the disease. With a price tag of over $2.5 While SMN1 gene mutations cause the disease, the number of copies of the SMN2 gene modifies the severity and helps determine the type of the condition.
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