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Children with rare diseases call for legislation
Children and teens with rare diseases candidly discussed their health challenges and legislation to enable new treatments during a Dec. 19 briefing for the Rare Disease Congressional Caucus.
“75% of us rare disease patients are children, and 30% of us do not live to see our fifth birthdays,” said 10-year-old Sati Cooper-McCann, whose Stickler syndrome lacks a cure, during the online briefing organized by the Rare Disease Legislative Advocates (RDLA) of the EveryLife Foundation for Rare Diseases.
“For the first time in a long time, I had hope,” said Rafaella Kopelan, 17, who described learning of a treatment for her ultra-rare blistering disease. “I could now dream about what I would do once the burdens have lessened. So, I let myself make friends.”
The legislation we need:
What’s next: Neither provision was included in the spending package that passed Congress at the end of the year, but the bills have bipartisan support, giving hope for action from the current Congress, which opened Friday.
See the Bio.News story and videos of the children’s testimony.
Buzz around JPM Week partnering bodes well for biotech dealmaking, BIO says
Increased interest in JPM Week this year is an indication of an improved dealmaking environment for biotech, say BIO experts facilitating partnering at the event.
What's happening: A record 6,000-plus meetings were scheduled through BIO’s partnering platform ahead of the J.P. Morgan Healthcare conference and related events in San Francisco.
Why it matters: “The macroeconomic indicators that we’re seeing include very strong interest in new deals—stronger than we’ve seen over the past two years,” says Bernard Fallon, BIO Deputy VP of Industry Programs. “We can expect a lot of activity at JPM Week.”
BIO’s cutting-edge partnering toolkit includes the BIO One-on-One Partnering™ platform, which lets registrants share profiles and describe their interests, to facilitate matchmaking and schedule meetings.
BIO also provides room to network, with more than 100 meeting tables at San Francisco’s Marriott Marquis. “For many BIO partners or people using our platform, our meeting space becomes kind of their hub,” says Mackensie Vernetti, BIO VP of Partnering.
The bottom line: “This can help them obtain the capital that will ultimately enable them to get new medicines to patients,” Fallon says.
Read more on Bio.News.
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