Rare diseases

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Cell and Gene Therapies in Rare Disorders: From Rarity to Recovery

The cell and gene therapy market has seen a revolutionary transition in recent years, with advancements in scientific research and novel methods of treatment driving a rise in development activities. This has resulted in an increase in the number of cell and gene therapy choices available to patients suffering from...

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Gene Therapy in Rare Disorders: Acceptance in Europe Faces Challenges

By bringing life-changing benefits to people with rare diseases and cancers, cell and gene therapies are reshaping the field of medicine. These treatments have recently gained tremendous publicity for meeting long-standing unmet needs and being extremely costly. But high production costs are limiting patient access...

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Biogen’s Aduhelm; FDA Approves Sanofi’s Enjaymo; NHS & Orchard Signs a Deal; Bristol’s Breyanzi & Abecma; Pharming’s Leniolisib; Eli Lilly’s Verzenio Trial; UCB’ Zilucoplan Trials Result; Eli Lilly’s Alzheimer’s Drug Donanemab

Biogen's Aduhelm Marketing and Approval are Under Scrutiny in New FTC and SEC Investigations Biogen can't seem to get a break when it comes to the Alzheimer's disease drug Aduhelm. Along with limited sales and a constraining Medicare coverage plan, the medicine is now facing additional scrutiny as part of a pair...

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Rare disease
Are Rare Diseases truly Rare?

Rare diseases, as the name depicts, affect only a small number of people compared to the other disease. Worldwide, one out of 15 persons could be affected by a rare disease, which is equivalent to approximately 400 million people. Most Rare diseases are often chronic, serious, and life-threatening. In the U...

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Pharma News
EicOsis receives USD 15M to fuel its non-opioid pain therapy; Alexion Pharmaceuticals buys Achillion Pharmaceuticals; Ipsen adds another drug to its rare disease pipeline

EicOsis has received USD 15Million grant from the National Institute of Drug Abuse (NIDA) to fund the clinical trials of non-opioid pain therapy. NIDA, with an aim to supplement NIH’s initiative Helping to End Addiction Long-Term (HEAL Initiative), has awarded the grant to the company. Deaths due to drug ov...

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Orphan drug development strategies

Introduction A disease that has not been adopted by the pharmaceutical industry because it provides little financial incentive for the private sector to make and market new medications to treat or prevent it.  An orphan disease may be a rare disease or a common disease that has been ignored because it is far more pr...

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