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- PMLiVE

bedaquiline

- PMLiVE

Sanofi expands rare disease pipeline with $2.2bn Inhibrx acquisition

The deal includes a mid-stage candidate for alpha-1 antitrypsin deficiency

Patient Advocate Engagement: A Customer Story

Learn how our client executed a series of 3 virtual touchpoints with 5 rare disease patient advocates to:- Develop a better understanding of the current treatment landscape and lived patient...

Impetus Digital

- PMLiVE

Johnson & Johnson’s bladder cancer drug Balversa granted full FDA approval

Urothelial carcinoma accounts for approximately 90% of bladder cancer cases

- PMLiVE

MHRA approves UCB’s Zilbrysq as generalised myasthenia gravis add-on therapy

The autoimmune disease has a global prevalence of 100 to 350 cases per every one million people

- PMLiVE

Takeda’s HyQvia granted FDA approval to treat rare neuromuscular disorder CIDP

Approximately five to seven people per 100,000 in the US are affected by the disorder

- PMLiVE

Johnson & Johnson to acquire ADC specialist Ambrx Biopharma for approximately $2bn

The deal includes an ADC candidate in clinical development to treat advanced prostate cancer

On a journey to diverse, inclusive, and more efficient rare disease trials

Although orphan drugs represent a sizeable portion of new drug approvals, most rare diseases still lack effective treatment. Even more so than for other conditions, rare disease management contains large care...

Impetus Digital

- PMLiVE

AstraZeneca and Ionis’ Wainua granted FDA approval for rare disease ATTRv-PN

ATTRv-PN leads to peripheral nerve damage with motor disability within five years of diagnosis

- PMLiVE

UK Biobank reveals new data from world’s largest genetic project

The data will accelerate the development of potentially lifesaving treatments and cures

- PMLiVE

Merck to acquire Caraway Therapeutics in a deal worth up to $610m

The deal gives Merck access to a pipeline of therapeutics for neurodegenerative and rare diseases

- PMLiVE

NICE recommends Chiesi’s alpha-mannosidosis enzyme replacement therapy

Alpha-mannosidosis, an extremely rare inherited condition characterised by a broad range of impairments, affects approximately 25 people in England

- PMLiVE

Janssen reveals positive results for nipocalimab in rheumatoid arthritis

About 13 million people worldwide are affected by the chronic inflammatory disease

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